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                Resources for Information | Approved Drugs

Resources for Information | Approved Drugs

                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
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          Resources for Information | Approved Drugs
          FDA D.I.S.C.O. Burst Edition: Tepmetko (tepotinib) and Ukoniq (umbralisib)Resources for Information | Approved Drugs FDA D.I.S.C.O. Burst Edition: Tepmetko (tepotinib) and Ukoniq (umbralisib)
  
  	
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Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide another quick update on two recent FDA cancer drug approvals.

On February 3, 2021, the FDA granted accelerated approval to tepotinib (brand name Tepmetko) for adult patients with metastatic non-small cell lung cancer (or NSCLC) harboring mesenchymal-epithelial transition (or MET) exon 14 skipping alterations. Efficacy was demonstrated in the VISION trial, a multicenter, non-randomized, open-label, multicohort study enrolling 152 patients with advanced or metastatic NSCLC with MET exon 14 skipping alterations. Patients received tepotinib 450 mg orally once daily until disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate determined by a blinded independent review committee using RECIST 1.1 and response duration. Among the 69 treatment naïve patients, the overall response rate was 43% with a median response duration of 10.8 months. Among the 83 previously treated patients, the overall response rate was 43% with a median response duration of 11.1 months. The most common adverse reactions occurring in more than 20% of patients were edema, fatigue, nausea, diarrhea, musculoskeletal pain, and dyspnea. Tepotinib can also cause interstitial lung disease, hepatotoxicity, and embryo-fetal toxicity.

This review used the Real-Time Oncology Review, which streamlined data submission prior to the filing of the entire clinical application, as well as the Oncology Center of Excellence Assessment Aid and the Product Quality Assessment Aid, voluntary submissions from the applicant to facilitate the FDA’s assessment.

The review was conducted under Project Orbis, which provides a framework for concurrent submission and review of oncology drugs among international partners. For this application, a modified Project Orbis was undertaken because of the timing of submission to other regulatory agencies.

On February 5, 2021 FDA granted accelerated approval to umbralisib (brand name Ukoniq), a kinase inhibitor including PI3K-delta and casein kinase CK1-epsilon, for the following indications:

Adult patients with relapsed or refractory marginal zone lymphoma who have received at least one prior anti-CD20-based regimen;
	And adult patients with relapsed or refractory follicular lymphoma who have received at least three prior lines of systemic therapy.Approval was based on two single-arm cohorts of an open-label, multi-center, multi-cohort trial, in 69 patients with marginal zone lymphoma who received at least one prior therapy, including an anti-CD20 containing regimen, and in 117 patients with follicular lymphoma after at least 2 prior systemic therapies.

Efficacy was based on overall response rate and duration of response using modified 2007 International Working Group criteria assessed by an independent review committee.

For patients with marginal zone lymphoma, the overall response rate was 49% with 16% achieving complete responses. Median duration of response was not reached in these patients. For patients with follicular lymphoma, the overall response rate was 43% with 3% achieving complete responses. Median duration of response was 11.1 months.

The prescribing information provides warnings and precautions for adverse reactions including infections, neutropenia, diarrhea and non-infectious colitis, hepatotoxicity, and severe cutaneous reactions.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatFDA.

Health care professionals should report serious adverse events to FDA’s MedWatch Reporting System at www.fda.gov/medwatch.

Follow the Division of Drug Information on Twitter @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. Thanks for tuning into the DISCO Burst Edition.

                                                                                  Content current as of:
      03/01/2021Resources for Information | Approved Drugs

          
                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
          Verified Clinical Benefit | Cancer Accelerated Approvals
          Withdrawn | Cancer Accelerated Approvals
          Other | Cancer Accelerated Approvals


                        Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description
 
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