Blueprint Medicines Corporation (NASDAQ:BPMC) Submits Supplementary New Drug Application For AYVAKIT™ In Advanced Systemic Mastocytosis

Blueprint Medicines Corporation (NASDAQ:BPMC) has submitted a supplemental New Drug Application to the US FDA for AYVAKIT™ (avapritinib) in treating advanced systemic mastocytosis (SM) patients. AYVAKIt is the company’s potent, selective D816B mutant KIT inhibitor that is under development for the treatment of non-advanced and advanced forms of SM.

Blueprint Medicines submits sNDA for AYVAKIT

The company requested a priority review for the sNDA and once granted it means a review could be in six months. The agency has a 60-day filing review period to establish if the application is complete and satisfactory for filing. AYVAKIT received a breakthrough therapy designation from the FDA for treating advanced SM which includes aggressive SM subtypes, as well as hematological neoplasm associated SM and mast cell leukemia.

Foudi Namouni, the company’s R&D president stated that the submission is a huge step for the company in the development of AYVAKIT for advanced systemic mastocytosis patients. Namouni said that the sNDA is based on exceptional clinical data set for the disease that demonstrated that patients that received AYVAKIT had high complete remission and overall response rates. They also showed prolonged survival and AYVAKIT was well tolerated in patients. Namouni added that they are looking forward to cooperating with the FDA during the review process to introduce the first precision therapy that targets SM’s underlying cause.

SM caused by KITD816V Mutation

SM is a rare, debilitating life-threatening disease driven by KIT D816V mutation. Normally uncontrolled activation and proliferation of mast cells can lead to severe, chronic, and unpredictable symptoms for SM patients. Most of those affected with non-advanced SM with devastating symptoms can experience a considerable, negative impact on the quality of life. A small number exhibit advanced SM encompassing a group of high-risk subtypes of SM which include aggressive SM, hematological neoplasm related SM, and mast cell leukemia.

Besides mast cell activation signs, advanced SM usually is related to organ damage because of mast cell infiltration as well as poor overall survival. Symptoms range from anaphylaxis, pruritis, maculopapular rash, brain fog, bone pain, and diarrhea which persists across the SM spectrum.