Blueprint Medicines Corporation (Nasdaq: BPMC), a US-based global precision therapy company, announced on Monday that it has received approval from the US Food and Drug Administration (FDA) for its AYVAKIT (avapritinib) intended for the treatment of adults with indolent systemic mastocytosis (ISM).

AYVAKIT will be available to patients with ISM in the United States at the recommended dose of 25mg once daily. It was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. AYVAKIT has been FDA approved for the treatment of advanced SM since June 2021.

The approval of AYVAKIT in ISM is based on data from the double-blind, placebo-controlled PIONEER trial – the largest study ever conducted for this disease – in which patients received AYVAKIT 25mg once daily plus best supportive care (AYVAKIT) or placebo plus best supportive care (placebo). AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden. AYVAKIT was well-tolerated with a favourable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity.

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.