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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: FDA approval of OGSIVEO (nirogacestat) for adult patients with progressing desmoid tumors who require systemic treatmentResources for Information | Approved Drugs FDA D.I.S.C.O. Burst Edition: FDA approval of OGSIVEO (nirogacestat) for adult patients with progressing desmoid tumors who require systemic treatment
  
  	
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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 a quick update on a recent FDA cancer drug approval.

On November 27, 2023, the FDA approved nirogacestat (brand name OGSIVEO) for adult patients with progressing desmoid tumors who require systemic treatment. This is the first approved treatment for desmoid tumors.

Efficacy was evaluated in DeFi an international, multicenter, randomized, double-blind, placebo-controlled trial in 142 patients with progressing desmoid tumors not amenable to surgery. Patients were eligible if the desmoid tumor had progressed within 12 months of screening. Patients were randomized to receive 150 mg nirogacestat or placebo orally twice daily until disease progression or unacceptable toxicity.

The major efficacy outcome measure was progression-free survival based on RECIST v1.1 as assessed by blinded independent central review or on clinical progression by the investigator and adjudicated by independent review. Median progression-free survival was not reached in the nirogacestat arm and 15.1 months in the placebo arm. An exploratory analysis of progression-free survival based on only radiographic progression demonstrated a hazard ratio of 0.31.

Objective response rate was an additional efficacy outcome measure. Objective response rate was 41% in the nirogacestat arm and 8% for those receiving placebo. Additionally, efficacy results were supported by change from baseline in patient-reported worst pain favoring the nirogacestat arm.

The most common adverse reactions were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.

This review used the Real-Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and 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 Drugs@FDA: FDA-Approved Drugs.

Health care professionals should report serious adverse events to MedWatch: The FDA Safety Information and Adverse Event Reporting Program.

Follow the Division of Drug Information on X (formally Twitter) @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. Thanks for tuning in to the D.I.S.C.O. Burst Edition.

                                                                                  Content current as of:
      01/08/2024Resources 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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