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                Resources for Information | Approved Drugs

Resources for Information | Approved Drugs

                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
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          Resources for Information | Approved Drugs
          FDA D.I.S.C.O. Burst Edition: FDA approval of Ayvakit (avapritinib) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemiaResources for Information | Approved Drugs FDA D.I.S.C.O. Burst Edition: FDA approval of Ayvakit (avapritinib) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia
  
  	
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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 have another quick update on a recent FDA cancer drug approval.

On June 16, 2021, the FDA approved avapritinib (brand name Ayvakit) for adult patients with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.

Efficacy was evaluated in EXPLORER and PATHFINDER, two multi-center, single-arm, open-label clinical trials enrolling patients with advanced systemic mastocytosis. The main efficacy outcome measure was overall response rate per modified IWG-MRT-ECNM criteria, as adjudicated by a central committee. Additional efficacy measures were duration of response, time to response, and changes in individual measures of mast cell burden.

The overall response rate in all patients evaluated in both trials combined was 57%, with 28% complete remissions and 28% partial remissions. The median duration of response was 38.3 months and the median time to response was 2.1 months.

The most common adverse reactions reported in more than 20% of patients with advanced systemic mastocytosis were edema, diarrhea, nausea, and fatigue or asthenia.

Avapritinib is not recommended for the treatment of patients with advanced systemic mastocytosis with platelet counts of less than 50 X 109/L.

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

Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program 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 and thanks for tuning in to the D.I.S.C.O. Burst Edition.

                                                                                  Content current as of:
      06/24/2021Resources 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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