RARE Daily

EC Approves Blueprint’s Ayvakyt for Adults with Certain GIST Tumors

September 28, 2020

Rare Daily Staff

The European Commission has granted Blueprint Medicines conditional marketing authorization for Ayvakyt as a monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumors harboring a specific mutation.

The approval is for the use of Ayvakyt to treat patients with GIST tumors with the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. It is the first approved therapy in Europe for patients with the specific mutation.

Ayvakyt is the first precision therapy approved in Europe to treat a genomically defined population of patients with GIST. The EC previously granted orphan medicinal product designation for Ayvakyt for the treatment of GIST. This U.S. Food and Drug Administration previously approve Ayvakyt for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

The EC approval is based on efficacy results from the phase 1 NAVIGATOR trial as well as combined safety results from the NAVIGATOR and phase 3 VOYAGER trials. Ayvakyt demonstrated deep and durable clinical activity and was generally well-tolerated in patients with PDGFRA D842V mutant GIST with or without prior therapy.

In 38 NAVIGATOR trial patients with PDGFRA D842V mutant GIST at a starting dose of 300 mg or 400 mg once daily, Ayvakyt  had an overall response rate of 95 percent, with 13 percent of patients achieving a complete response, and the median duration of response was 22.1 months. The median progression-free survival was 24 months, and the median overall survival was not reached.

The most frequently reported adverse reactions (≥20 percent) were nausea, fatigue, anemia, periorbital edema, face edema, hyperbilirubinemia, diarrhea, vomiting, peripheral edema, increased lacrimation, decreased appetite and memory impairment.

“Ayvakyt is the first highly active treatment option for PDGFRA D842V mutant GIST, offering much-needed hope for this rare patient population who have long been underserved by existing therapies,” said Jeff Albers, CEO of Blueprint Medicines. “As we progress toward our Ayvakyt  launches in Europe, we are focused on raising awareness about the therapy’s differentiated clinical profile and the importance of mutational testing, and working closely with health authorities to enable patient access to Ayvakyt as rapidly as possible.”

In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany following the EC approval, and the timing of Ayvakyt availability will vary for other countries based on local reimbursement and access pathways. As part of the conditional marketing authorization, Blueprint Medicines plans to conduct an observational, long-term study in patients with PDGFRA D842V mutant GIST treated with Ayvakyt.

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