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June 16, 2021Ayvakit Receives New Indication for Rare Mast Cell Disorders
June 16, 2021 – The U.S. FDA has approved a new indication for AyvakitTM (avapritinib), manufactured by Blueprint Medicines. The drug is now indicated to treat adults who have advanced systemic mastocytosis (AdvSM), including aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
AdvSM is a group of rare disorders affecting the mast cells – a type of immune cell found in connective tissue throughout the body. In AdvSM, the body produces too many mast cells. These excess mast cells accumulate in areas such as the skin, bone marrow, and other organs of the body. This can cause a wide range of signs and symptoms, including inflammation, itching, hives, gastrointestinal problems, anemia, bleeding disorders, pain in the bones and muscles, depression, loss of bone density, and organ damage. AdvSM is associated with a shorter life expectancy compared to people who do not have the disease.
In clinical trials, Ayvakit delivered an overall response rate of 57%, with 28% of patients achieving complete remission of AdvSM and an additional 28% of patients achieving partial remission. The median time to treatment response was 2.1 months. Duration of response was 38.3 months.
Recommended dosing under the new indication is 200mg taken by mouth once daily until disease progression or unacceptable toxicity occur. Ayvakit should be taken on an empty stomach at least one hour before and two hours after a meal.
Ayvakit first received FDA approval in 2020, to treat adults diagnosed with unresectable or metastatic gastrointestinal stromal tumor harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.