CHMP positive for Ayvakyt (avapritinib) to treat indolent systemic mastocytosis . -Blueprint Medicines

Systemic mastocytosis (SM) 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. Most patients rely on polypharmacy of best supportive care medications for the management of symptoms, which often are inadequately controlled. Ayvakyt was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease and has the potential to help the estimated 40,000 people with ISM in the European Union move beyond multiple, symptom-directed therapies.

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

The CHMP based its positive opinion on data from the registrational double-blind, placebo-controlled PIONEER trial – the largest study ever conducted for this disease – in which patients received Ayvakyt 25 mg or placebo once daily plus best supportive care. As previously reported in NEJM Evidence,(previously cited) Ayvakyt demonstrated clinically meaningful improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

"In Europe, patients with ISM currently have no approved therapies to target the underlying driver of disease, and the need to advance treatment beyond supportive care has never been more critical," said PD Dr. Frank Siebenhaar, Head of University Outpatient Clinic at Charité Institute of Allergology in Berlin. "As a physician who specializes in treating and researching ISM, I have seen firsthand the significant impact ISM has on the daily lives of patients and their families. The PIONEER trial results demonstrate the potential to improve quality of life and transform the standard of care for the ISM community.