Avapritinib Receives Support for Indolent Systemic Mastocytosis Indication in Europe

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive recommendation for avapritinib in adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms not controlled by therapies, according to avapritinib manufacturer, Blueprint Medicines Corporation.

“Indolent systemic mastocytosis is often characterized by severe symptoms, significant morbidities and high healthcare resource utilization, with a profound impact on patients and their families,” stated Becker Hewes, MD, Chief Medical Officer of Blueprint Medicines, in a press release.

Patients with systemic mastyocytosis (SM) are primarily treated with several symptom-directed medications, but many patients still experience symptoms. Avapritinib is a potent, selective inhibitor of KIT D816V, the primary driver of SM. If approved by the European Commission (EC), avapritinib will be the first treatment directed at the primary disease driver approved for the approximately 40,000 patients with ISM in Europe, according to the release.

“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 Frank Siebenhaar, MD, Head of University Outpatient Clinic at Charité Institute of Allergology in Berlin.

Currently, avapritinib is approved in the United States for adults with ISM and advanced SM. Avapritinib is also approved by the EMA for the treatment of advanced SM and PDGFRA D842V-mutated gastrointestinal stromal tumors.

The positive recommendation from the CHMP was based on the pivotal PIONEER trial, which reported once-daily avapritinib was well-tolerated with favorable safety signals and predominantly mild adverse events. The EC will review the CHMP recommendation, and a final decision on the ISM indication is expected in Q1 2024.