On March 25, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that the European Commission (EC) has expanded the current indication for AYVAKYT (avapritinib) to include monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy (Press release, Blueprint Medicines, MAR 25, 2022, View Source [SID1234610999]).

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"Today we are incredibly proud to bring an innovative new treatment option to individuals who have been impacted by advanced systemic mastocytosis," said Georg Pirmin Meyer, M.D., Senior Vice President, International at Blueprint Medicines. "We believe that AYVAKYT has the potential to shift the treatment paradigm to a precision therapy approach in advanced forms this disease, and we look forward to working closely with national reimbursement bodies across Europe to bring AYVAKYT to patients."

In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany following the EC approval, and the timing of AYVAKYT commercial availability will vary for other countries based on local reimbursement and access pathways. AYVAKYT will be available in 25 mg, 50 mg, 100mg and 200mg dose strengths, and the recommended starting dose in advanced SM is 200 mg once daily.

The EC decision follows the positive opinion by the Committee for Medicinal Products for Human Use (CHMP) and is based on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across all disease subtypes after at least one systemic therapy and a generally well-tolerated safety profile.

About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation and for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy. Under the brand name AYVAKIT, the medicine is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL).1

It is also approved under the brand name AYVAKIT in Mainland China for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and in Hong Kong and Taiwan for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.2-4

AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the European Union, UK, U.S., or Greater China, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKYT/AYVAKIT globally for the treatment of advanced and non-advanced SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis. The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at [email protected] and +31 85 064 4001. Additional information is available at blueprintclinicaltrials.com and clinicaltrials.gov.

Please click here to see the Summary of Product Characteristics for AYVAKYT.

About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. Across advanced SM subtypes, the median overall survival is approximately 3.5 years in ASM, approximately two years in SM-AHN and less than six months in MCL.5 In Europe, there are about 40,000 patients with SM, and advanced SM represents about 5 to 10 percent of this patient population.6

Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Historically, there had been no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.7,8