Blueprint Medicines submits supplemental NDA to the FDA for Ayvakit for the treatment of advanced systemic mastocytosis.

Blueprint Medicines Corporation announced the submission of a supplemental new drug application (sNDA) to the FDA for Ayvakit (avapritinib) for the treatment of adult patients with advanced systemic mastocytosis (SM). Ayvakit is a potent and selective inhibitor of D816V mutant KIT, the primary driver of SM, and is being developed to treat advanced and non-advanced forms of the disease. Blueprint Medicines requested priority review for this application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine whether the sNDA is complete and acceptable for filing. The FDA granted breakthrough therapy designation to Ayvakit for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia.