According to a recent article in Biospace, patients with advanced systemic mastocytosis (SM) can turn to a newly FDA-approved treatment by Blueprint Medicines called Ayvakit (avapritinib). The drug is the first of its kind to be available for this hematologic disease, which causes extensive damage to organ systems. The list of disorders targeted by Ayvakit includes aggressive SM, mast cell leukemia, and SM with corresponding hematologic growth of tissue (neoplasm).
About Advanced SM
The KIT D816V mutation has been found in the peripheral blood of nearly all advanced SM patients. One characteristic of SM is the uncontrolled growth of immune cells called mast cells. These mast cells build up in the bones and intestines or under the skin. The results are gastrointestinal problems and abnormal spots or bumps on the skin. Most SM patients do not survive longer than four years.
The Phase 1 EXPLORER and Phase 2 PATHFINDER Trials
Ayvakit’s second regulatory approval in two years was founded on data submitted to the FDA from the EXPLORER AND PATHFINDER studies. Results of these trials provided evidence of Ayvakit’s clinical efficacy in advanced SM subtypes, whether or not the patient had received prior treatment.
At the eleventh month, Ayvakit had a fifty-seven percent response rate with a median duration of thirty-eight months.
Looking Forward
Ayvakit may be established as a new standard of care for advanced mastocytosis patients and an excellent example of precision therapy. (The full article is available here).
