Avyakit From Blueprint Medicines Secures Approval for Treating Rare Organ Diseases

The FDA has approved Blueprint Medicines’ Ayvakit (avapritinib) for treating adults with advanced systemic mastocytosis (SM), a group of rare diseases in which white blood cells accumulate in various organs.

Advanced cases are marked by widescale damage to multiple organ systems, including the GI tract, spleen, lymph nodes and bone marrow.

The approval of avapritinib, a kinase inhibitor, was supported by results from two clinical trials in which treated patients had an overall response of 57 percent, including 28 percent complete remissions and 28 percent partial remissions.

The FDA has granted Ayvakit Priority Review, Breakthrough Therapy and Orphan Drug designations.