Blueprint's Ayvakyt Receives Positive CHMP Opinion For Indolent Systemic Mastocytosis Treatment

(RTTNews) - Blueprint Medicines Corp. (BPMC) announced the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency or EMA has issued a positive opinion recommending the approval of Ayvakyt or avapritinib for the treatment of adult patients with indolent systemic mastocytosis or ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment.

The CHMP opinion was based on the positive PIONEER trial, in which once-daily AYVAKYT achieved significant improvements across a broad range of symptoms with a comparable safety profile to placebo.

The CHMP opinion will now be reviewed by the European Commision and a final decision to expand Ayvakyt's indication to include ISM is expected in the first-quarter 2024.

Currently, Ayvakyt is approved by the EMA for the treatment of advanced Systemic mastocytosis and PDGFRA D842V-mutant gastrointestinal stromal tumors.

Systemic mastocytosis is a rare hematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients' quality of life.

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