CENTER FOR DRUG EVALUATION AND RESEARCH

INNOVATION PREDICTABILITY ACCESS

January 2024

Advancing Health Through Innovation: 

New Drug Therapy 

Approvals 2023
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 ii

Table of Contents

Director’s Message ������������������������������������������������������1

Executive Summary ��������������������������������������������������� 2

Innovation Across Medical Conditions ���������������������������������������������������������������������2

New Drugs for Patients with Rare Diseases�������������������������������������������������������������3

Efficiencies in Bringing Therapies to Market ������������������������������������������������������������4

CDER’s Novel Drug Approvals of 2023�������������������������� 5

First-in-Class Drugs��������������������������������������������������������������������������������������������������6

Drugs for Rare Diseases ������������������������������������������������������������������������������������������7

Other Novel Drug Approvals ������������������������������������������������������������������������������������8

Innovation: Use of Expedited Development 

and Review Pathways ������������������������������������������������10

Fast Track���������������������������������������������������������������������������������������������������������������10

Breakthrough Therapy�������������������������������������������������������������������������������������������� 11

Priority Review�������������������������������������������������������������������������������������������������������� 11

Accelerated Approval����������������������������������������������������������������������������������������������12

Overall Use of Expedited Development and Review Methods�������������������������������12

Predictability: Meeting PDUFA Goals��������������������������� 13

Access: First Cycle Approvals 

and First in U.S. Approvals�����������������������������������������

New Uses of Approved Drugs �������������������������������������16

Approved Drugs Expanded 

for New Pediatric Populations �����������������������������������18

Biosimilar Approvals ����������������������������������������������� 20

Conclusion��������������������������������������������������������������� 24

Appendix A: CDER’s Novel Approvals of 2023 ������������� 25

Appendix B: Novel Drug Designations ������������������������ 29
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 1

Director’s Message

Welcome to FDA’s Center for Drug Evaluation and Research’s (CDER) 13th annual report, 

Advancing Health Through Innovation: New Drug Therapy Approvals. This report highlights 

the role that CDER plays in bringing safe and effective drugs to patients and consumers.

2023 was an important year for public health. Perhaps most notably, after more than three 

years, the U.S. Department of Health and Human Services declaration of a Public Health 

Emergency (PHE) for COVID-19 ended in May. While the PHE has ended, CDER is continuing to review drug applications for the treatment of COVID-19 infections.To that point, 

CDER approved the first oral antiviral pill for COVID-19 in 2023 and is supporting further 

drug development in this space.

Our 2023 New Drug Therapy Approvals Report highlights CDER’s “novel” drug approval 

actions — which are for therapies that have not previously been approved in the U.S. The 

report covers other important CDER actions as well, such as expanding the use or patient 

population of previously approved drugs. The report also describes drugs approved in new 

dosage forms or formulations.

I want to particularly point out our efforts in the opioid overdose space. In 2023, CDER 

took actions to make three opioid overdose reversal drugs available without a prescription, 

which can increase access to life-saving therapies. We also approved new dosage forms 

of drugs that reverse opioid overdose and treat opioid use disorder. Our efforts align with 

FDA’s Overdose Prevention Framework, which aims to prevent drug overdose and reduce 

overdose-related death

But our work is not confined to any one area of drug development, and our 2023 drug 

approvals collectively target a wide range of diseases and conditions. Many of the 2023 

approvals are for patients with few or no treatment options, including those with rare diseases. Other drug approvals offer improvements in effectiveness, safety, or ease of use 

compared to standard-of-care therapies. 

We approved the majority of the 2023 therapies on or before their goal dates, or congressionally authorized agreements with industry. We also approved most of these drugs in the 

U.S. before our regulatory counterparts did so in other countries. 

In encouraging news for biosimilars, CDER approved five biosimilars, including three biosimilars for reference products that did not have a corresponding biosimilar. To date, CDER 

has approved 45 biosimilars for 14 reference products.

This report captures CDER’s 2023 approvals. FDA’s Center for Biologics Evaluation and 

Research (CBER) also approves important biologics and CDER and CBER may collaborate on approval actions. Please visit CBER’s webpage for 2023 Biological Approvals for 

information on CBER’s 2023 product approvals.

We hope this report showcases CDER’s steadfast commitment to protecting and advancing 

patient care through the careful and vigorous review and approval of meaningful treatments.

Patrizia Cavazzoni, M.D. 

Director, Center for Drug Evaluation and Research

Patrizia Cavazzoni, M.D.

Director, Center for Drug 

Evaluation and Research
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 2

Executive Summary 

CDER approved a variety of safe and effective drug therapies in 2023. These approvals, 

spanning a broad scope of diseases and conditions, aim to help many people live better and 

potentially longer lives.

Innovation Across Medical Conditions 

In 2023, we approved 55 new drugs never before approved or marketed in the U.S., known 

as “novel” drugs. We also made other important approval decisions, such as expanding the 

use or patient population of previously approved drugs. 

The 2023 actions, both novel and other important drug approvals, focus on prevention, diagnosis, and treatment of diseases and conditions such as:

• Infectious diseases, including COVID-19, respiratory syncytial virus (RSV), hospital-acquired and ventilator-associated bacterial pneumonia, and HIV-1.

• Neurological conditions, such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and migraine.

• Opioid use, misuse, and abuse. 

• Heart, blood, kidney, and endocrine diseases, including type 2 diabetes in pediatrics, 

types of anemia, pediatric hormone deficiency, and chronic weight management.

• Lung diseases, such as asthma and cystic fibrosis.

• Gastrointestinal conditions, including inflammatory bowel disease and pediatric functional constipation.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 3

• Different types of cancers, such as colorectal, prostate, lung, and low-grade gliomas 

(tumors that start in the brain).

• Women’s health, such as postpartum depression, hot flashes due to menopause, and 

nonprescription oral contraception. 

New Drugs for Patients with Rare Diseases

Patients with rare diseases are often in critical need of new therapies, as these individuals generally have few or no existing treatment options. In 2023, 28 of 55, or 51% of our 

novel drug approvals received orphan drug designation because they target rare diseases, 

including:*

• Friedreich’s ataxia, an inherited, degenerative disease that damages the nervous system.

• Candidemia and invasive candidiasis, which are serious and life-threatening fungal 

infections

• Rett syndrome, a genetic, neurological disorder that affects brain development.

• CD55-deficient protein-losing enteropathy (CHAPLE disease), a genetic disease that 

affects the immune system.

• Paroxysmal nocturnal hemoglobinuria, a disease that causes red blood cells to break 

apart.

• Activated phosphoinositide 3-kinase delta, a genetic disorder that impairs the immune 

system.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 4

In 2023, CDER also approved many therapies for rare cancers or tumors, including:

• Mantle cell lymphoma, an aggressive form of non-Hodgkin’s lymphoma.

• Nasopharyngeal carcinoma, a rare head and neck cancer.

• Large B-cell lymphoma.

• Desmoid tumors, noncancerous growths in the connective tissue.

*Not all drugs for rare diseases necessarily receive orphan designation.

Efficiencies in Bringing Therapies to Market

Our 2023 approvals demonstrate efficiencies in our review process, as shown by the 

following:

• User Fee Goals Performance: Of the 55 new drugs approved in 2023, CDER met 

or exceeded its Prescription Drug User Fee Act (PDUFA) goal dates for 49 of these 

approvals (89%). A number of novel drug approvals in 2023 were delayed due to 

COVID-19 related foreign travel restrictions, which hindered onsite inspections within 

the user fee review timeline.

• First Cycle Approvals: In 2023, CDER approved 46 of the 55 novel approvals (84%) 

on the first cycle. This differs from when CDER initially is unable to approve a drug 

because information in the application does not support approval. Subsequently, the 

sponsor resubmits the application with additional information, starting another review 

cycle that may lead to drug approval.

• Approvals in U.S. Before Other Countries: 35 of the 55 novel drugs approved in 

2023 (64%) were first approved in the U.S.

• Expedited Programs for Serious Conditions: CDER has four broadly applicable 

programs to facilitate and expedite development and review of drugs for serious or 

life-threatening conditions: Fast Track designation, Breakthrough Therapy designation, Priority Review designation, and Accelerated Approval. In 2023, 36 of the 55 of 

CDER’s novel drug approvals (65%) used one or more of these expedited programs, 

which helped bring new therapies to the market sooner.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 5

CDER’s Novel Drug Approvals of 2023

In 2023, CDER approved 55 novel drugs, either as new molecular entities (NMEs) under New 

Drug Applications (NDAs), or as new therapeutic biologics under Biologics License Applications (BLAs). The active ingredient(s) in a novel drug have not been approved in the U.S.

CDER’s novel drug approvals for 2023 are listed alphabetically below by trade name.*

See Appendix B for 

a summary chart 

of designations 

for CDER’s novel 

approvals. 

Trade Name Active Ingredient(s)

Agamree vamorolone

Aphexda motixafortide

Augtyro repotrectinib

Beyfortus nirsevimab-alip

Bimzelx bimekizumab-bkzx

Brenzavvy bexagliflozin

Columvi glofitamab-gxbm

Daybue trofinetide

Defencath taurolidine, heparin

Elfabrio pegunigalsidase alfa-iwxj

Elrexfio elranatamab-bcmm

Epkinly epcoritamab-bysp

Exxua gepirone

Fabhalta iptacopan

Filspari sparsentan

Filsuvez birch triterpenes

Fruzaqla fruquintinib

Inpefa sotagliflozin

Izervay avacincaptad pegol

Jaypirca pirtobrutinib

Jesduvroq daprodustat

Joenja leniolisib

Lamzede velmanase alfa-tycv

Leqembi lecanemab-irmb

Litfulo ritlecitinib

Loqtorzi toripalimab-tpzi

Miebo perfluorohexyloctane

Ngenla somatrogon-ghla

Ogsiveo nirogacestat

Ojjaara momelotinib

Trade Name Active Ingredient(s)

Omvoh mirikizumab-mrkz

Orserdu elacestrant

Paxlovid nirmatrelvir, ritonavir (co-packaged)

Pombiliti cipaglucosidase alfa-atga

Posluma flotufolastat F 18

Qalsody tofersen

Rezzayo rezafungin

Rivfloza nedosiran

Rystiggo rozanolixizumab-noli

Ryzneuta efbemalenograstim alfa-vuxw

Skyclarys omaveloxolone

Sohonos palovarotene

Talvey talquetamab-tgvs

Truqap capivasertib

Vanflyta quizartinib

Velsipity etrasimod

Veopoz pozelimab-bbfg

Veozah fezolinetant

Wainua eplontersen

Xacduro sulbactam, durlobactam (co-packaged)

Xdemvy lotilaner

Zavzpret zavegepant

Zilbrysq zilucoplan

Zurzuvae zuranolone

Zynyz retifanlimab-dlwr

*This information is accurate as of December 31, 2023. In 

rare instances, CDER may need to change a drug’s NME 

designation or the status of its application as a novel BLA. 

For instance, new information may become available that 

could lead to a reconsideration of the original designation or 

status. If CDER makes these types of changes, the agency 

intends to communicate the nature of, and the reason for, any 

revisions as appropriate.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 6

CDER’s Annual Novel Drug Approvals: 2014–2023

The 10-year graph below shows that from 2014 through 2023, CDER has averaged about 

46 novel drug approvals per year.

First-in-Class Drugs

CDER identified 20 of the 55 novel drugs approved (36%) in 2023 as first-in-class. These 

drugs have mechanisms of action different from those of existing therapies.

Novel drugs approved in 2023 that CDER identified as first-in-class were: 

Daybue, Defencath, Fabhalta, Filspari, Filsuvez, Jesduvroq, Joenja, Lamzede, Miebo, 

Ogsiveo, Paxlovid, Qalsody, Rivfloza, Skyclarys, Sohonos, Talvey, Truqap, Veopoz, 

Veozah, Xdemvy 

Notable examples of novel first-in-class approvals include: 

• Daybue (trofinetide) oral solution as the first treatment for patients aged two years 

and older with Rett syndrome, a rare, genetic neurological disorder that affects brain 

development.

• Jesduvroq (daprodustat) tablets as the first oral treatment for anemia (decreased 

number of red blood cells) caused by chronic kidney disease for adults receiving dialysis for at least four months.

• Miebo (perfluorohexyloctane) ophthalmic solution to treat signs and symptoms of dry 

eye disease, which occurs when a patient’s tears do not provide sufficient eye lubrication, leading to potential ocular (eye-related) inflammation and damage. 

• Paxlovid (nirmatrelvir and ritonavir, co-packaged for oral use) tablets to treat 

mild-to-moderate COVID-19 in adults who are at high risk for progression to severe 

COVID-19, including hospitalization or death. Paxlovid is the first oral antiviral pill 

approved to treat COVID-19 in adults.

CDER’s Novel Drug Approvals By Year

First-in-Class 

Drugs

36%

CDER identified 20 out of 

the 55 novel drugs (36%) 

approved in 2023 as 

first-in-class.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 7

• Skyclarys (omaveloxolone) capsules as the first treatment for Friedreich’s ataxia, a 

rare, inherited, degenerative disease that damages the nervous system, characterized 

by impaired coordination and difficulty walking.

• Talvey (talquetamab-tgvs) injection to treat adults with refractory (treatment-resistant) 

or relapsed (recurring) multiple myeloma who have received other therapies. It was 

approved through the Accelerated Approval pathway. 

• Veozah (fezolinetant) tablets to treat moderate to severe vasomotor symptoms, or hot 

flashes, due to menopause.

• Xdemvy (lotilaner) ophthalmic solution as the first drug to treat Demodex blepharitis, 

chronic eyelid inflammation caused by Demodex folliculorum, a microscopic mite.

Drugs for Rare Diseases

In 2023, 28 of CDER’s 55 novel drug approvals (51%) received orphan drug designation 

because they target rare diseases (diseases that affect fewer than 200,000 people in 

the U.S.). Patients with rare diseases often have few or no drugs available to treat their 

conditions

Novel drugs approved in 2023 with the orphan drug designation were:*

Agamree, Aphexda, Augtyro, Daybue, Elrexfio, Fabhalta, Filspari, Filsuvez, Jaypirca, 

Joenja, Lamzede, Loqtorzi, Ngenla, Ogsiveo, Ojjaara, Pombiliti, Qalsody, Rezzayo, 

Rivfloza, Rystiggo, Skyclarys, Sohonos, Talvey, Vanflyta, Veopoz, Wainua, Zilbrysq, Zynyz

Examples of novel approvals of 2023 for rare diseases include:

• Fabhalta (iptacopan) tablets as the first oral treatment for paroxysmal nocturnal 

hemoglobinuria, a disease that causes red blood cells to break apart. 

• Filspari (sparsentan) tablets to reduce proteinuria (elevated protein in the urine) in 

adults with primary immunoglobulin A (IgA) nephropathy at risk of rapid disease progression. IgA nephropathy is a chronic kidney disease that can lead to kidney failure. 

Filspari was approved through the Accelerated Approval pathway.

• Jaypirca (pirtobrutinib) tablets to treat relapsed or refractory mantle cell lymphoma, 

an aggressive form of non-Hodgkin lymphoma, after receiving other therapies. Later 

in 2023, CDER approved Jaypirca to treat adults with chronic lymphocytic leukemia 

or small lymphocytic lymphoma after receiving other therapies. Both indications were 

approved through the Accelerated Approval pathway.

• Joenja (leniolisib) tablets as the first treatment for activated phosphoinositide 

3-kinase delta syndrome (APDS) in patients 12 years and older. APDS is a genetic disorder that impairs the immune system.

• Lamzede (velmanase alfa) injection to treat non-central nervous system manifestations of alpha-mannosidosis, a rare genetic condition in which patients do not have 

the alpha-mannosidase enzyme. Symptoms include intellectual disability, hearing loss, 

weakened immune system, distinctive facial features, skeletal abnormalities, and muscle weakness.

Drugs for Rare 

Diseases

51%

More than half (28) of the 

drugs CDER approved in 

2023 received orphan drug 

designation.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 8

• Loqtorzi (toripalimab-tpzi) infusion to treat recurrent or metastatic (spreading) nasopharyngeal carcinoma (NPC) when used together with or following other therapies. 

NPC is a rare head and neck cancer. 

• Ogsiveo (nirogacestat) tablets to treat desmoid tumors, which are noncancerous 

growths in the connective tissue, such as the abdomen, arms, and legs.

• Ojjaara (momelotinib) tablets to treat myelofibrosis, a type of bone marrow cancer 

that causes scar tissue buildup in the bone marrow, disrupting the body’s normal production of blood cells. 

• Rezzayo (rezafungin) injection to treat candidemia and invasive candidiasis, which 

are serious and life-threatening fungal infections, in patients 18 years or older with limited or no alternative treatments.

• Rystiggo (rozanolixizumab-noli) injection for adults with the two most common 

subtypes of generalized myasthenia gravis, a chronic autoimmune neuromuscular condition that causes muscle weakness and severe fatigue.

• Sohonos (palovarotene) capsules to reduce the volume of new heterotopic ossification (bone formation outside the skeleton) in females eight years and older and males 

10 years and older with fibrodysplasia ossificans progressiva, a disorder in which 

bones gradually form in muscle tissue and connective tissue.

• Veopoz (pozelimab) injection as the first drug to treat CHAPLE disease, a very 

rare genetic disease. CHAPLE disease affects the immune system and can be 

life-threatening

• Wainua (eplontersen) injection to treat hereditary transthyretin-mediated amyloidosis, 

a genetic disorder that leads to organ and tissue dysfunction. 

*Not all drugs for rare diseases necessarily receive orphan designation.

Other Novel Drug Approvals 

In addition to the first-in-class and drugs for rare diseases, CDER approved these notable 

approvals in 2023:

• Beyfortus (nirsevimab-alip) injection as the first approved drug to prevent RSV lower 

respiratory tract disease in all (i.e., not only high-risk) babies born during or entering 

their first RSV season and certain high-risk children up to 24 months. While most 

patients experience mild cold-like symptoms, some infants, especially with their first 

RSV infection, develop lower respiratory tract disease such as pneumonia and bronchiolitis (swelling of the lungs’ small airway passages).

• Columvi (glofitamab-gxbm) injection to treat types of relapsed or refractory diffuse 

large B-cell lymphoma (DLBCL) and high-grade B‐cell lymphoma. DLBCL is marked

by rapid tumor growth in the lymph nodes, spleen, liver, bone marrow, or other tissues 

and organs. Columvi was approved through the Accelerated Approval pathway. 

• Defencath (taurolidine and heparin) catheter lock solution to reduce catheter-related 

bloodstream infections in adults with kidney failure who are receiving chronic hemodialysis (a procedure to purify the blood) through a central venous catheter.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 9

• Epkinly (epcoritamab-bysp) injection to treat types of relapsed or refractory DLBCL 

and high-grade B‐cell lymphoma. Epkinly was approved through the Accelerated

Approval pathway.

• Fruzaqla (fruquintinib) capsules to treat refractory, metastatic colorectal cancer. 

• Izervay (avacincaptad pegol) injection to treat geographic atrophy secondary to age-related macular degeneration (AMD). Geographic atrophy is an advanced form of AMD 

that leads to progressive and irreversible retinal cell loss and permanent vision loss.

• Leqembi (lecanemab-irmb) injection to treat Alzheimer’s disease, a progressive neurologic disorder that is the most common cause of dementia. Leqembi was approved 

through the Accelerated Approval pathway and later converted to traditional approval 

after clinical benefit was confirmed, both in 2023. 

• Orserdu (elacestrant) tablets to treat breast cancer with certain genetic features and 

disease progression after at least one line of endocrine therapy. 

• Xacduro (sulbactam and durlobactam) injection to treat hospital-acquired bacterial 

pneumonia and ventilator-associated bacterial pneumonia caused by Acinetobacter 

baumannii-calcoaceticus complex.

• Zavzpret (zavegepant) nasal spray to treat acute migraine with or without aura (a 

migraine warning sign). 

• Zurzuvae (zuranolone) tablets as the first oral medication to treat postpartum depression, a complex mix of physical, emotional, and behavioral changes that can occur 

around childbirth.

CDER approved the 

first oral medication 

for postpartum 

depression in 2023.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 10

Innovation: Use of Expedited Development 

and Review Pathways

CDER used diverse regulatory approaches to enhance and expedite drug review in 2023. 

These approaches enable increased flexibility, efficiency, and interactions between CDER 

staff and drug developers. They often also allow shorter review times to speed the availability of new therapies to patients with serious conditions, especially in cases where there 

are no satisfactory alternatives, while preserving FDA’s rigorous standards for safety and 

effectiveness

Fast Track

CDER granted Fast Track status to 25 of the 55 novel drugs (45%) in 2023. Fast Track speeds 

development and review of new drugs and biologics by increasing the level of communication 

between FDA and drug developers and by enabling CDER to review portions of a drug application on a rolling basis.

Drugs granted Fast Track status were:

Agamree, Beyfortus, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Filsuvez, Fruzaqla, 

Izervay, Jaypirca, Lamzede, Leqembi, Ogsiveo, Orserdu, Paxlovid, Qalsody, 

Rezzayo, Skyclarys, Truqap, Vanflyta, Veopoz, Xacduro, Zurzuvae, Zynyz

Fast Track 

45%

CDER designated 25 of 

the 55 novel drugs (45%) 

as Fast Track.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 11

Breakthrough Therapy

CDER designated 9 of the 55 novel drugs (16%) in 2023 as Breakthrough Therapies. A Breakthrough Therapy designation includes all the Fast Track program features and offers intensive 

FDA guidance during drug development, including involvement from senior managers.

Drugs designated with Breakthrough Therapy status were: 

Elrexfio, Fabhalta, Izervay, Leqembi, Loqtorzi, Ogsiveo, Pombiliti, Rivfloza, Talvey

Priority Review

In 2023, 31 of the 55 novel drugs approved (56%) were designated Priority Review. A drug 

receives Priority Review if CDER determines that the drug treats a serious condition and, if 

approved, would provide a significant improvement in safety or effectiveness of the treatment, 

diagnosis, or prevention of the serious condition. Generally speaking, a Priority Review application is one on which CDER aims to take action within six months of filing (compared to a 

target date of 10 months under standard review).

(In some instances, sponsors may redeem a priority review voucher under CDER’s Priority 

Review Voucher program. Such drugs are not included in the list below, and do not meet 

Priority Review criteria.)

Drugs designated Priority Review were:

Augtyro, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Epkinly, Filspari, Filsuvez, 

Fruzaqla, Izervay, Jaypirca, Joenja, Lamzede, Leqembi, Loqtorzi, Ogsiveo, Orserdu, 

Paxlovid, Qalsody, Rezzayo, Rystiggo, Skyclarys, Sohonos, Talvey, Truqap, Vanflyta, 

Veopoz, Xacduro, Zurzuvae, Zynyz

Breakthrough 

Therapy

16%

CDER identified 9 of the 

55 novel drugs (16%) 

approved in 2023 as 

Breakthrough Therapies.

Priority Review 

56%

31 of the 55 drugs (56%) 

approved in 2023 were 

designated as Priority 

Review. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 12

Accelerated Approval

CDER approved 9 of the 55 novel drugs (16%) in 2023 under the Accelerated Approval 

pathway. This program aims to bring drugs to market that can provide treatment for unmet 

medical needs on a faster timeline than would be possible following a traditional approval 

pathway. Accelerated Approval may be an option for a new drug intended to treat a serious 

condition that offers a meaningful advantage over available therapies. For drugs eligible to 

follow the Accelerated Approval pathway, a determination of safety and effectiveness may 

be made based not on measures of direct clinical benefit, but rather on one of two alternative endpoints: (1) a surrogate endpoint that is reasonably likely to predict clinical benefit; or 

(2) an intermediate clinical endpoint that is reasonably likely to predict clinical benefit. Use 

of such endpoints may enable the drug to be studied for a shorter treatment duration and 

receive Accelerated Approval based on these findings. Importantly, however, for products 

approved under the Accelerated Approval pathway, FDA requires post-approval studies 

designed to confirm clinical benefit, and, among other things, may withdraw an accelerated 

approval product from the market for failure to confirm clinical benefit.

The novel drugs approved via Accelerated Approval were: 

Columvi, Elrexfio, Epkinly, Filspari, Jaypirca, Leqembi,* Qalsody, Talvey, Zynyz

*Was converted to traditional approval in 2023 as well.

Overall Use of Expedited Development and Review Methods

Thirty-six of the 55 novel drug approvals of 2023 (65%) used one or more expedited programs, specifically Fast Track designation, Breakthrough Therapy designation, Priority 

Review designation, or Accelerated Approval.

Novel drugs approved in 2023 that used at least one expedited program were: 

Agamree, Augtyro, Beyfortus, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Epkinly, 

Fabhalta, Filspari, Filsuvez, Fruzaqla, Izervay, Jaypirca, Joenja, Lamzede, Leqembi, Loqtorzi, 

Ogsiveo, Orserdu, Paxlovid, Pombiliti, Qalsody, Rezzayo, Rivfloza, Rystiggo, Skyclarys, 

Sohonos, Talvey, Truqap, Vanflyta, Veopoz, Xacduro, Zurzuvae, Zynyz

Accelerated Approval

16%

CDER identified 9 of the 

55 novel drugs (16%) as 

Accelerated Approvals. 

CDER used at least 

1 expedited program 

to speed approval 

of 65% of all novel 

drugs approved in 

2023.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 13

Predictability: Meeting PDUFA Goals

Under PDUFA, industry is assessed user fees that provide resources to CDER to expand 

capabilities for review activities. With PDUFA, applications are reviewed targeting specific 

timeframes. Throughout 2023, CDER met or exceeded the PDUFA goal date for taking 

action on 89% (49 of 55) of the novel drugs approved. 

Novel drugs approved in 2023 on or before their PDUFA goal dates were: 

Agamree, Aphexda, Augtyro, Beyfortus, Brenzavvy, Columvi, Daybue, Defencath, Elfabrio, 

Elrexfio, Epkinly, Exxua, Fabhalta, Filspari, Filsuvez, Fruzaqla, Inpefa, Izervay, Jaypirca, 

Jesduvroq, Joenja, Lamzede, Leqembi, Litfulo, Miebo, Ogsiveo, Ojjaara, Omvoh, Orserdu, 

Paxlovid, Posluma, Qalsody, Rezzayo, Rivfloza, Skyclarys, Sohonos, Talvey, Truqap, 

Vanflyta, Velsipity, Veopoz, Veozah, Wainua, Xacduro, Xdemvy, Zavzpret, 

Zilbrysq, Zurzuvae, Zynyz

Meeting PDUFA Goals

89%

In 2023, 49 out of the 55 

novel drugs (89%) were 

approved on or before 

their PDUFA goal date. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 14

Access: First Cycle Approvals 

and First in U.S. Approvals

First Cycle Approvals

CDER approved 46 of the 55 novel drugs of 2023 (84%) on the “first cycle” of review. This 

high percentage is in part reflective of the extent to which CDER staff provide clarity to drug 

developers on the necessary study design elements and other data needed in the drug 

application to support a full and comprehensive drug assessment. 

Novel drugs approved in 2023 on the first cycle were:

Agamree, Aphexda, Augtyro, Beyfortus, Brenzavvy, Columvi, Daybue, Elrexfio, Epkinly, 

Fabhalta, Filspari, Fruzaqla, Inpefa, Izervay, Jaypirca, Jesduvroq, Joenja, Lamzede, 

Leqembi, Litfulo, Miebo, Ogsiveo, Ojjaara, Orserdu, Paxlovid, Pombiliti, Posluma, Qalsody, 

Rezzayo, Rivfloza, Rystiggo, Ryzneuta, Skyclarys, Talvey, Truqap, Vanflyta, Velsipity, 

Veopoz, Veozah, Wainua, Xacduro, Xdemvy, Zavzpret, Zilbrysq, Zurzuvae, Zynyz

First Cycle Approvals

84%

In 2023, CDER approved 

46 of the 55 novel drugs 

(84%) on the first cycle.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 15

Approval in the U.S. Before Other Countries

Thirty-five of the 55 novel drugs approved in 2023 (64%) were approved in the U.S. before 

any other country.

Novel drugs of 2023 approved first in the U.S. were:

Agamree, Aphexda, Augtyro, Brenzavvy, Daybue, Elrexfio, Epkinly, Exxua, Fabhalta, 

Filspari, Izervay, Jaypirca, Joenja, Leqembi, Litfulo, Ogsiveo, Ojjaara, Orserdu, Posluma, 

Qalsody, Rezzayo, Rivfloza, Rystiggo, Skyclarys, Talvey, Truqap, Velsipity, Veopoz, 

Veozah, Wainua, Xacduro, Xdemvy, Zavzpret, Zurzuvae, Zynyz

64% of the novel 

drugs approved in 

2023 were approved 

in the U.S. before 

any other country. 

First in the U.S.

64%

35 of the 55 novel drugs 

approved in 2023 were 

first approved in the U.S.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 16

New Uses of Approved Drugs 

After CDER approves a new treatment, a drug sponsor may generate new data about the 

product that suggests an additional use. The drug sponsor may then submit an application 

to modify or expand the use of an approved drug based on this new data. 

The products below are some 2023 CDER approvals for new uses or indications of an 

approved drug:

• Ayvakit (avapritinib) tablets were first approved in 2020 to treat types of gastrointestinal stromal tumors. Ayvakit was approved in 2023 for indolent systemic mastocytosis, 

a rare disorder that results in the buildup of too many mast cells (a type of white blood 

cell).

• Eylea (aflibercept) injection was originally approved in 2011 for neovascular (wet) 

age-related macular degeneration. In 2023, CDER approved Eylea to treat retinopathy 

of prematurity (ROP), an eye disease that can occur in babies born prematurely. In 

ROP, abnormal blood vessels grow in the retina and can lead to vision loss.

• Ilaris (canakinumab) injection was originally approved in 2009. In 2023, CDER 

approved Ilaris to treat gout flares in adults in whom certain other therapies are not 

advised. Gout is a type of arthritis characterized by sudden attacks of pain, swelling, 

and redness in one or more joints. 

• Jemperli (dostarlimab-gxly) injection was initially approved in 2021. In 2023, CDER 

approved Jemperli for two endometrial cancer uses, as a single agent and in combination with other therapies. Endometrial cancer occurs in the tissues of the endometrium, 

which is the lining of the uterus.

• Kevzara (sarilumab) injection was first approved in 2017 for moderately to severely 

active rheumatoid arthritis. In 2023, CDER approved it for adults with polymyalgia 

rheumatica (PMR) who did not adequately respond to corticosteroids or who cannot 

tolerate corticosteroid taper (gradual dose reduction). PMR is an inflammatory disorder 

that causes muscle pain and stiffness around the shoulders and hips.

• Keytruda (pembrolizumab) injection was first approved in 2014. In 2023, CDER 

approved Keytruda for several new uses, including in combination with Padcev for 

patients with locally advanced or metastatic urothelial carcinoma who are not eligible 

for a type of chemotherapy; for types of non-small cell lung cancer as an adjuvant 

(after other therapies) therapy; and for early-stage non-small cell lung cancer as a neoadjuvant (before other therapies) and adjuvant treatment. 

• Linzess (linaclotide) capsules were first approved in 2012. In 2023, CDER approved 

it as the first treatment for pediatric functional constipation in patients six to 17 years. 

Pediatric functional constipation occurs when patients have infrequent bowel movements with hard stools that can be difficult or painful to pass.

• Lonsurf (tipiracil hydrochloride and trifluridine) tablets were first approved in 2015. 

In 2023, CDER approved Lonsurf, alone or in combination with another therapy (bevacizumab), for patients with previously treated metastatic colorectal cancer. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 17

• Lynparza (olaparib) tablets were first approved in 2014. In 2023, Lynparza was

approved in combination with another therapy for adults with a type of prostate cancer

that is metastatic and castration-resistant (i.e., keeps growing when the amount of testosterone is reduced to very low levels).

• Padcev (enfortumab vedotin-ejfv) injection was first approved in 2019 as a single

agent. CDER approved Padcev in combination with Keytruda in 2023 to treat adults

with locally advanced or metastatic urothelial cancer.

• Polivy (polatuzumab vedotin-piiq) injection was first approved in 2019 to treat adults

with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, after at least two therapies. In 2023, CDER expanded the indication for adults with 

previously untreated DLBCL, not otherwise specified, or high-grade B-cell lym-phoma 

that is considered low-intermediate risk or higher.

• Rexulti (brexpiprazole) tablets were initially approved in 2015 as an add-on therapy

for major depressive disorder and as a treatment for schizophrenia. In 2023, CDER

approved Rexulti as the first drug to treat agitation associated with dementia due to

Alzheimer’s disease.

• Rinvoq (upadacitinib) tablets were originally approved in 2019 for adults with moderately to severely active rheumatoid arthritis. In 2023, CDER approved Rinvoq for adults

with moderately to severely active Crohn’s disease with an inadequate response or

intolerance to other therapies (specifically TNF blockers). Rinvoq is the first approved

oral product for moderately to severely active Crohn’s disease, a chronic disease that

causes inflammation in the digestive tract.

• Talzenna (talazoparib) capsules were first approved in 2018 to treat types of breast

cancer. In 2023, Talzenna was approved in combination with another therapy to treat a

type of metastatic castration-resistant prostate cancer.

• Tukysa (tucatinib) tablets were first approved in 2020. In 2023, CDER approved

Tukysa for a type of colorectal cancer that has progressed following certain

chemotherapies.

• Verzenio (abemaciclib) tablets were initially approved in 2017. In 2023, CDER

approved Verzenio with endocrine therapy (tamoxifen or an aromatase inhibitor) for a

type of early breast cancer with a high risk of recurrence.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 18

Approved Drugs Expanded 

for New Pediatric Populations 

Section 505B of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (often referred to 

by the legislation that originally created it, the Pediatric Research Equity Act, or PREA) and 

section 505A of the FD&C Act (often referred to by the legislation that originally created it, 

the Best Pharmaceuticals for Children Act, or BPCA) give CDER the authority to require 

(PREA) or request (BPCA) pediatric studies under certain circumstances. These two laws 

have been largely responsible for the inclusion of pediatric information in the labeling for 

many drugs. 

Upon drug approval, CDER may require pediatric studies of that drug under PREA. In 

response to that requirement, sponsors may submit new data to support the safe and effective use of the drug in the pediatric population studied. Sponsors submit this data in an 

application to expand the patient population. Under BPCA, sponsors may obtain additional 

marketing exclusivity for pediatric studies requested in a Pediatric Written Request.

The products below are examples of 2023 approvals for drugs expanded to include 

new pediatric populations:

• Cresemba (isavuconazonium sulfate) injection was initially approved in 2015 to 

treat invasive aspergillosis and invasive mucormycosis, which are fungal diseases. 

In 2023, CDER extended Cresemba’s patient population to include patients one year 

and older

• Evkeeza (evinacumab-dgnb) injection was initially approved in 2021 as an add-on to 

other therapies to treat patients 12 years and older with homozygous familial hypercholesterolemia, a genetic disease that causes extremely high cholesterol levels. 

Evkeeza’s patient population was extended to individuals five years and older in 2023. 

• Jardiance (empagliflozin) tablets were approved in 2014 as an addition to diet 

and exercise to improve blood sugar in adults with type 2 diabetes. In 2023, CDER 

expanded the patient population to children 10 years and older. 

• Livmarli (maralixibat) oral solution was approved in 2021 to treat cholestatic 

pruritus (extreme itching) in patients one year and older with Alagille syndrome, a 

genetic disease affecting the liver, heart, kidneys, and other organs. In 2023, CDER 

expanded the patient population to babies three months and older. 

• Mekinist (trametinib) tablets were first approved in 2013 and are now approved for 

various oncology indications in combination with Tafinlar [see page 19]. In 2023, the 

patient population was extended to one year and older. 

• Opdivo (nivolumab) injection was originally approved in 2014 and has subsequently 

been approved for certain oncology indications. In 2023, CDER extended the patient 

population to 12 years and older for certain indications.

• Revatio (sildenafil citrate) tablets had been approved to treat a type of pulmonary 

arterial hypertension (PAH) in adults. In 2023, CDER expanded the population to 

include pediatric patients. PAH is a rare, progressive disorder characterized by high 

blood pressure in the lungs’ arteries. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 19

• Rozlytrek (entrectinib) capsules were first approved in 2019 for several oncology 

indications, including solid tumors with a NTRK gene fusion (where two genes form 

one hybrid gene) in patients 12 years and older. In 2023, CDER extended the patient 

population for this indication down to one month. CDER has also approved a new 

oral pellet formulation for Rozlytrek. 

• Synjardy (empagliflozin and metformin hydrochloride) tablets were first approved 

in 2015 as an addition to diet and exercise to improve blood sugar in adults with type 

2 diabetes whose disease meets certain criteria. CDER extended the patient population to children 10 years and older in 2023. 

• Tafinlar (dabrafenib) tablets were first approved in 2013 and are now approved for 

various oncology indications in combination with Mekinist [see page 18]. In 2023, the 

patient population was extended to one year and older. 

• Takhzyro (lanadelumab-flyo) injection was initially approved in 2018 to prevent 

attacks of hereditary angioedema, a disorder characterized by recurrent episodes of 

severe swelling, in patients 12 years and older. CDER expanded the patient population to two years and older in 2023. 

• Trikafta (elexacaftor, tezacaftor, and ivacaftor) tablets were first approved in 2019 

to treat cystic fibrosis in patients 12 years and older. In 2023, CDER extended the 

patient population down to two years. 

• Triumeq Pd (abacavir, dolutegravir, and lamivudine) tablets for oral suspension 

were first approved in 2022 to treat HIV-1 infection in adults and children who weigh 

at least 10 kg. In 2023, the pediatric population was extended down to children who 

are at least three months old and weigh at least six kg.

• Yervoy (ipilimumab) injection was first approved in 2011. In 2023, CDER expanded 

the population to include patients 12 years and older to treat unresectable or metastatic melanoma in combination with Opdivo [see page 18]

• Zinplava (bezlotoxumab) injection was initially approved in 2016 to reduce recurrence of Clostridioides difficile infection (CDI) in patients 18 years or older receiving 

antibacterial drug treatment at a high risk for recurrence. In 2023, CDER expanded 

the patient population to children one year and older. CDI is a bacteria-caused infection that results in colon inflammation.

In 2023, CDER 

approved a new 

class of medicines 

to treat pediatric 

type 2 diabetes. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 20

Biosimilar Approvals 

The biosimilar pathway is an abbreviated approval pathway for biologics that are highly 

similar to and have no clinically meaningful differences from an FDA-approved biological reference product. This pathway was established to provide more treatment options, 

increase patient access, and potentially reduce the cost of therapies through competition. 

In 2023, CDER approved five new biosimilars for five reference products, including three 

reference products that did not have a corresponding biosimilar. Several drugs were 

approved as interchangeable biosimilars, which are biosimilars that may be substituted for 

the reference product at the pharmacy similar to how generics are substituted, subject to 

state law

• Avzivi (bevacizumab-tnjn) injection was approved to treat types of metastatic colorectal cancer and hepatocellular carcinoma (reference product: Avastin)

• Tofidence (tocilizumab-bavi) injection was approved to treat types of arthritis, including rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile 

idiopathic arthritis (reference product: Actemra)

• Tyruko (natalizumab-sztn) injection was approved as a monotherapy (a single therapy) to treat relapsing forms of multiple sclerosis and to induce and maintain a clinical 

response in adults with moderately to severely active Crohn’s disease whose condition 

meets certain criteria (reference product: Tysabri)

• Wezlana (ustekinumab-auub) injection was approved to treat plaque psoriasis and 

psoriatic arthritis in patients six years and older, and Crohn’s disease and ulcerative 

colitis in adults (reference product: Stelara). Wezlana was approved as an interchangeable biosimilar. 

In 2023, CDER 

approved 5 

biosimilars for 5 

reference products. 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 21

• Yuflyma (adalimumab-aaty) injection was approved to treat a variety of inflammatory 

conditions (reference product: Humira)

In 2023, CDER also approved notable changes to the following biosimilars:

• Abrilada (adalimumab-afzb) injection was originally approved in 2019 for several 

inflammatory conditions. In 2023, CDER approved it as an interchangeable biosimilar 

(reference product: Humira)

• Byoovis (ranibizumab-nuna) injection was initially approved in 2021 to treat certain 

eye conditions. In 2023, CDER approved it as an interchangeable biosimilar (reference 

product: Lucentis)

CDER has approved a total of 45 biosimilars for 14 different reference products since 2015. 

This includes at least one biosimilar for each of these top selling biologics in the U.S.: nine 

biosimilars to Humira; six biosimilars to Neulasta; five biosimilars to Herceptin and Avastin; 

four biosimilars to Remicade; three biosimilars to Rituxan and Neupogen; two biosimilars to 

Lantus, Enbrel, and Lucentis; and one biosimilar to Epogen/Procit, Actemra, Stelara, and 

Tysabri. Multiple biosimilars for an approved reference product can enhance competition, 

which may lead to reduced costs for both patients and our health care system.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 22

Other Important Approvals

New formulations of approved drugs can offer significant therapeutic advances. Similarly, new dosage forms (such as from a capsule to a chewable tablet for those unable 

to swallow pills) can help increase adherence, make sure patients take the proper dose, 

and improve quality of life for patients who must use the medication on a prolonged basis. 

Making a drug available as an over-the-counter product can also increase patient access 

to therapies. Below are examples of new formulations, new dosage forms, over-the-counter 

actions, and other notable approval actions of 2023:

• Airsupra (albuterol and budesonide) inhalation aerosol for as-needed treatment or 

prevention of bronchoconstriction (tightening of lung airways) and to reduce the risk 

of asthma attacks in adults. Airsupra is a combination product of two approved active 

ingredients, albuterol and budesonide.

• Akeega (niraparib and abiraterone acetate) tablets to treat a type of prostate cancer 

that meets certain disease criteria, together with prednisone (a steroid). Akeega is a 

combination product of two approved active ingredients, niraparib and abiraterone. 

• Brixadi (buprenorphine) extended-release injection to treat moderate-to-severe opioid use disorder. Brixadi is now approved in both weekly and monthly under-the-skin 

injectable formulations at varying doses, including lower doses that may be appropriate 

for those who do not tolerate higher doses of extended-release buprenorphine.

• Entyvio (vedolizumab) injection was approved in 2014 for intravenous (into the vein) 

administration to treat moderately to severely active ulcerative colitis and Crohn’s disease in adults. In 2023, CDER approved Entyvio as an under-the-skin injection to treat 

moderately to severely active ulcerative colitis in certain adults. This will allow patients 

to self-administer the medication after training, avoiding needing to go to an infusion 

center

• Hepzato (melphalan hydrochloride) injection. Melphalan, the active ingredient, has 

been approved for different oncology indications. In 2023, CDER approved Hepzato 

with the same active ingredient to treat liver metastases (metastatic growths) in certain 

patients with uveal melanoma, a rare cancer that develops in a part of the eye. 

• Lampit (nifurtimox) tablets were converted from Accelerated Approval to full approval 

for use in pediatric patients from birth to younger than 18 years to treat Chagas 

disease (American Trypanosomiasis) caused by the Trypanosoma cruzi parasite. 

Chagas disease can cause heart, digestive, and neurological problems and may be 

life-threatening.

• Lodoco (colchicine) tablets. Lodoco, with the previously approved active ingredient 

colchicine, was approved in 2023 to reduce the risk of certain cardiovascular events in 

adults with established atherosclerotic disease (thickening of the arteries) or with multiple risk factors for cardiovascular disease.

• Mydcombi (tropicamide and phenylephrine hydrochloride) ophthalmic spray, 

a combination of two approved active ingredients, was approved in 2023 to induce 

mydriasis (pupil dilation) for diagnostic procedures and in other conditions.

In 2023, CDER 

converted Lampit 

tablets from 

Accelerated 

Approval to full 

approval to treat 

Chagas disease.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 23

• Narcan (naloxone hydrochloride) nasal spray had been approved as a prescription 

drug to reverse the effects of opioid overdose. In 2023, CDER approved Narcan as a 

nonprescription drug. 

• Opill (norgestrel) tablets had been approved as a prescription drug to prevent 

pregnancy. In 2023, CDER approved Opill as the first nonprescription daily oral 

contraceptive. 

• Opvee (nalmefene hydrochloride) nasal spray was approved in a new dosage form 

in 2023 to reverse the effects of opioid overdose. Nalmefene hydrochloride (the active 

ingredient) had been approved as an injection drug. 

• Prevymis (letermovir) tablets and injection were first approved in 2017 to prevent 

cytomegalovirus (CMV) infection and disease in adults at high risk for CMV who 

received an allogeneic (from a donor) hematopoietic stem cell transplant. In 2023, 

CDER approved Prevymis for the same indication in adults at high risk for CMV who 

received a kidney transplant. 

• RiVive (naloxone hydrochloride) nasal spray was approved in 2023 as a nonprescription drug to reverse the effects of opioid overdose. Its active ingredient, naloxone 

hydrochloride, had been approved for prescription use.

• Ryzumi (phentolamine) ophthalmic solution was approved in 2023 to treat pharmacologically induced mydriasis (eye dilation). CDER had previously approved its active 

ingredient, phentolamine.

• Syfovre (pegcetacoplan) injection. Pegcetacoplan, the active ingredient, was first 

approved as an under-the-skin injection in 2021. In 2023, CDER approved Syfovre for 

intravitreal use (a shot directly into the eye) to treat geographic atrophy resulting from 

AMD

• Technegas (kit for preparation of technetium Tc 99m labeled carbon) inhalation 

aerosol was approved as a radioactive diagnostic agent for visualization of lung ventilation and evaluation of pulmonary embolism (a blockage in the pulmonary arteries), 

when paired with perfusion imaging. Other dosage forms of technetium Tc 99m have 

been previously approved. 

• Zepbound (tirzepatide) injection. In 2023, CDER approved Zepbound, with the previously approved active ingredient tirzepatide, for chronic weight management, in 

addition to a reduced-calorie diet and increased physical activity, in adults with obesity 

or overweight and at least one weight-related comorbid condition. 

Please note that all drugs carry risks and patients should review the drug labeling and 

consult with their health care professional to determine their preferred course of treatment.
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 24

Conclusion

Reviewing a drug application — whether for a novel drug or a supplemental approval — is a 

collaborative, well-coordinated process that involves scientific, regulatory, and policy experts 

from throughout CDER and sometimes other parts of the agency. For each application, we 

perform a very careful and diligent analysis of safety and effectiveness data, including a benefit-risk analysis that factors in the severity of the disease or condition, the currently available 

treatment options, and the intended patient population. If the therapy meets the standard 

for approval, we must reach agreement on the indication, labeling, safety issues, and other 

considerations

We often consult outside scientific experts, patients and patient advocates, industry 

representatives, academics, and other community members who are involved in drug 

development and review. Each of these parties has their unique expertise and perspective, and we consider their viewpoints. We take our regulatory decision-making seriously, 

because we know our decisions affect the health and well-being of patients and consumers 

nationwide
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 25

Appendix A: CDER’s Novel Approvals of 2023 

(in alphabetical order)

For information about vaccines, allergenic products, blood and blood products, cellular and 

gene therapy products, go to the 2023 Biologics License Application Approvals.

Approval 

Date

Proprietary 

Name

Active Ingredient(s) Summary of FDA-approved 

use on approval date

(see Drugs@FDA for complete 

indication)

Dosage Form

10/26/2023 Agamree vamorolone To treat Duchenne muscular 

dystrophy

Oral suspension 

9/8/2023 Aphexda motixafortide To use with filgrastim to mobilize 

hematopoietic stem cells to 

peripheral blood for collection 

and subsequent autologous 

transplantation in patients with 

multiple myeloma

Injection 

11/15/2023 Augtyro repotrectinib To treat ROS1-positive non-small cell 

lung cancer

Capsule

7/17/2023 Beyfortus nirsevimab-alip To prevent respiratory syncytial virus 

(RSV) lower respiratory tract disease

Injection

10/17/2023 Bimzelx bimekizumab-bkzx To treat moderate-to-severe plaque 

psoriasis 

Injection

1/20/2023 Brenzavvy bexagliflozin To improve glycemic control in type 2 

diabetes mellitus as an adjunct to diet 

and exercise

Tablet

6/15/2023 Columvi glofitamab-gxbm To treat diffuse large B-cell 

lymphoma, not otherwise specified 

or large B-cell lymphoma arising from 

follicular lymphoma, after two or 

more lines of systemic therapy

Injection

3/10/2023 Daybue trofinetide To treat Rett syndrome Oral solution 

11/15/2023 Defencath taurolidine, heparin To prevent catheter-related 

bloodstream infection in patients 

on dialysis using a central venous 

catheter

Catheter lock 

solution

5/9/2023 Elfabrio pegunigalsidase alfa-iwxj To treat confirmed Fabry disease Injection 

8/14/2023 Elrexfio elranatamab-bcmm To treat relapsed or refractory 

multiple myeloma after at least four 

lines of therapy

Injection

5/19/2023 Epkinly epcoritamab-bysp To treat relapsed or refractory diffuse 

large B-cell lymphoma, not otherwise 

specified, and high-grade B-cell 

lymphoma after two or more lines of 

systemic therapy

Injection

9/22/2023 Exxua gepirone To treat major depressive disorder Tablet 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 26

Appendix A (continued)

Approval 

Date

Proprietary 

Name

Active Ingredient(s) Summary of FDA-approved 

use on approval date

(see Drugs@FDA for complete 

indication)

Dosage Form

12/5/2023 Fabhalta iptacopan To treat paroxysmal nocturnal 

hemoglobinuria

Capsule 

2/17/2023 Filspari sparsentan To reduce proteinuria in primary 

immunoglobulin A nephropathy at 

risk of rapid disease progression

Tablet 

12/18/2023 Filsuvez birch triterpenes To treat wounds associated 

with dystrophic and junctional 

epidermolysis bullosa

Gel

11/8/2023 Fruzaqla fruquintinib To treat refractory metastatic 

colorectal cancer

Capsule

5/26/2023 Inpefa sotagliflozin To reduce the risk of cardiovascular 

death, hospitalization for heart 

failure, and urgent heart failure visits 

Tablet

8/4/2023 Izervay avacincaptad pegol To treat geographic atrophy 

secondary to age-related macular 

degeneration

Intravitreal 

solution

1/27/2023 Jaypirca pirtobrutinib To treat relapsed or refractory mantle 

cell lymphoma after at least two lines 

of systemic therapy, including a BTK 

inhibitor 

Tablet

2/1/2023 Jesduvroq daprodustat To treat anemia caused by chronic 

kidney disease

Tablet

3/24/2023 Joenja leniolisib To treat activated phosphoinositide 

3-kinase delta syndrome

Tablet

2/16/2023 Lamzede velmanase alfa-tycv To treat non-central nervous system 

manifestations of alpha-mannosidosis

Injection 

1/6/2023 Leqembi lecanemab-irmb To treat Alzheimer's disease Injection 

6/23/2023 Litfulo ritlecitinib To treat severe alopecia areata Capsule 

10/27/2023 Loqtorzi toripalimab-tpzi To treat recurrent or metastatic 

nasopharyngeal carcinoma with or 

following other therapies

Injection 

5/18/2023 Miebo perfluorohexyloctane To treat signs and symptoms of dry 

eye disease

Ophthalmic 

solution

6/27/2023 Ngenla somatrogon-ghla To treat growth failure due to 

inadequate secretion of endogenous 

growth hormone

Injection 

11/27/2023 Ogsiveo nirogacestat To treat desmoid tumors Tablet

9/15/2023 Ojjaara momelotinib To treat intermediate or high-risk 

myelofibrosis

Tablet 

10/26/2023 Omvoh mirikizumab-mrkz To treat ulcerative colitis Injection
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 27

Appendix A (continued)

Approval 

Date

Proprietary 

Name

Active Ingredient(s) Summary of FDA-approved 

use on approval date

(see Drugs@FDA for complete 

indication)

Dosage Form

1/27/2023 Orserdu elacestrant To treat certain types of advanced or 

metastatic breast cancer with disease 

progression following at least one 

line of endocrine therapy

Tablet

5/25/2023 Paxlovid nirmatrelvir, ritonavir 

(co-packaged)

To treat mild-to-moderate COVID-19 

at high risk for progression to severe 

COVID-19

Tablet 

9/28/2023 Pombiliti cipaglucosidase alfa-atga To treat late-onset Pompe disease 

with miglustat 

Injection 

5/25/2023 Posluma flotufolastat F 18 To use with positron emission 

tomography for prostate cancer 

imaging

Injection

4/25/2023 Qalsody tofersen To treat a form of amyotrophic lateral 

sclerosis

Injection 

3/22/2023 Rezzayo rezafungin To treat candidemia and invasive 

candidiasis

Injection 

9/29/2023 Rivfloza nedosiran To lower urinary oxalate levels in 

primary hyperoxaluria type 1 and 

relatively preserved kidney function

Injection

6/26/2023 Rystiggo rozanolixizumab-noli To treat generalized myasthenia 

gravis

Injection 

11/16/2023 Ryzneuta efbemalenograstim alfavuxwTo decrease the incidence of 

infection, as manifested by febrile 

neutropenia, associated with 

myelosuppressive anti-cancer drugs

Injection

2/28/2023 Skyclarys omaveloxolone To treat Friedreich's ataxia Capsule 

8/16/2023 Sohonos palovarotene To reduce the volume of new 

heterotopic ossification in 

fibrodysplasia ossificans progressiva

Capsule 

8/9/2023 Talvey talquetamab-tgvs To treat relapsed or refractory 

multiple myeloma after at least four 

therapies

Injection 

11/16/2023 Truqap capivasertib To treat breast cancer that meets 

certain disease criteria

Tablet

7/20/2023 Vanflyta quizartinib To use as part of a treatment regimen 

for newly diagnosed acute myeloid 

leukemia that meets certain criteria

Tablet 

10/12/2023 Velsipity etrasimod To treat moderately to severely active 

ulcerative colitis 

Tablet 

8/18/2023 Veopoz pozelimab-bbfg To treat CD55-deficient proteinlosing enteropathy (PLE) (i.e., CHAPLE 

disease)

Injection 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 28

Appendix A (continued)

Approval 

Date

Proprietary 

Name

Active Ingredient(s) Summary of FDA-approved 

use on approval date

(see Drugs@FDA for complete 

indication)

Dosage Form

5/12/2023 Veozah fezolinetant To treat moderate to severe hot 

flashes caused by menopause

Tablet 

12/21/2023 Wainua eplontersen To treat hereditary transthyretinmediated amyloidosisInjection

5/23/2023 Xacduro sulbactam, durlobactam 

(co-packaged)

To treat hospital-acquired bacterial 

pneumonia and ventilator-associated 

bacterial pneumonia caused by 

susceptible isolates of Acinetobacter 

baumannii-calcoaceticus complex

Injection 

7/24/2023 Xdemvy lotilaner To treat Demodex blepharitis Ophthalmic 

solution

3/9/2023 Zavzpret zavegepant To treat migraine Nasal spray 

10/17/2023 Zilbrysq zilucoplan To treat generalized myasthenia 

gravis

Injection 

8/4/2023 Zurzuvae zuranolone To treat postpartum depression Capsule 

3/22/2023 Zynyz retifanlimab-dlwr To treat metastatic or recurrent locally 

advanced Merkel cell carcinoma

Injection 
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 29

Appendix B: Novel Drug 

Designations

(in alphabetical order)

Trade Name

Fast First First in 

Agamree ✓ ✓ ✓ ✓ ✓

Aphexda ✓ ✓ ✓ ✓

Augtyro ✓ ✓ ✓ ✓ ✓

Beyfortus ✓ ✓ ✓

Bimzelx

Brenzavvy ✓ ✓ ✓

Columvi ✓ ✓ ✓ ✓ ✓

Daybue ✓ ✓ ✓ ✓ ✓ ✓ ✓

Defencath ✓ ✓ ✓ ✓

Elfabrio ✓ ✓ ✓

Elrexfio ✓ ✓ ✓ ✓ ✓ ✓ ✓ ✓

Epkinly ✓ ✓ ✓ ✓ ✓

Exxua ✓ ✓

Fabhalta ✓ ✓ ✓ ✓ ✓ ✓

Filspari ✓ ✓ ✓ ✓ ✓ ✓ ✓

Filsuvez ✓ ✓ ✓ ✓ ✓

Fruzaqla ✓ ✓ ✓ ✓

Inpefa ✓ ✓

Izervay ✓ ✓ ✓ ✓ ✓ ✓

Jaypirca ✓ ✓ ✓ ✓ ✓ ✓ ✓

Jesduvroq ✓ ✓ ✓

Joenja ✓ ✓ ✓ ✓ ✓ ✓

Lamzede ✓ ✓ ✓ ✓ ✓

Leqembi ✓ ✓ ✓ ✓ ✓ ✓ ✓

Litfulo ✓ ✓ ✓

Loqtorzi ✓ ✓ ✓

Miebo ✓ ✓ ✓

First-in-Class

Orphan

Fast Track

Breakthrough Therapy

Priority Review

Accelerated Approval

PDUFA Goal Met

First Cycle Approval

First in the United States
Advancing Health Through Innovation: New Drug Therapy Approvals 2023 30

Appendix B (continued)

Trade Name

Ngenla ✓

Ogsiveo ✓ ✓ ✓ ✓ ✓ ✓ ✓ ✓

Ojjaara ✓ ✓ ✓ ✓

Omvoh ✓

Orserdu ✓ ✓ ✓ ✓ ✓

Paxlovid ✓ ✓ ✓ ✓ ✓

Pombiliti ✓ ✓ ✓

Posluma ✓ ✓ ✓

Qalsody ✓ ✓ ✓ ✓ ✓ ✓ ✓ ✓

Rezzayo ✓ ✓ ✓ ✓ ✓ ✓

Rivfloza ✓ ✓ ✓ ✓ ✓ ✓

Rystiggo ✓ ✓ ✓ ✓

Ryzneuta ✓

Skyclarys ✓ ✓ ✓ ✓ ✓ ✓ ✓

Sohonos ✓ ✓ ✓ ✓

Talvey ✓ ✓ ✓ ✓ ✓ ✓ ✓ ✓

Truqap ✓ ✓ ✓ ✓ ✓ ✓

Vanflyta ✓ ✓ ✓ ✓ ✓

Velsipity ✓ ✓ ✓

Veopoz ✓ ✓ ✓ ✓ ✓ ✓ ✓

Veozah ✓ ✓ ✓ ✓

Wainua ✓ ✓ ✓ ✓

Xacduro ✓ ✓ ✓ ✓ ✓

Xdemvy ✓ ✓ ✓ ✓

Zavzpret ✓ ✓ ✓

Zilbrysq ✓ ✓ ✓

Zurzuvae ✓ ✓ ✓ ✓ ✓

Zynyz ✓ ✓ ✓ ✓ ✓ ✓ ✓

First-in-Class

Orphan

Fast Track

Breakthrough Therapy

Priority Review

Accelerated Approval

PDUFA Goal Met

First Cycle Approval

First in the United States
U.S. Food and Drug Administration

www.fda.gov

Center for Drug Evaluation and Research 

U.S. Food and Drug Administration 

10903 New Hampshire Avenue

Silver Spring, Maryland 20903