The US Food and Drug Administration (FDA) has approved avapritinib (Ayvakit) for the treatment of adults with indolent systemic mastocytosis.
Systemic mastocytosis is a rare disease, usually caused by mutations the KIT D816V gene. The disorder is characterized by uncontrolled mast cell proliferation and activation across multiple organ systems. Indolent systemic mastocytosis accounts for approximately 90% of systemic mastocytosis. Common symptoms may include anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain. Most adults present with skin lesions, usually in the form of urticaria pigmentosa.
In the video clip below, Brenton Mar, MD, PhD, Senior Medical Director at Blueprint Medicines, gives an overview of systemic mastocytosis and advanced systemic mastocytosis.
Avapritinib is the first medicine indicated for indolent systemic mastocytosis. The tyrosine kinase inhibitor is also approved to treat advanced systemic mastocytosis and gastrointestinal stomal tumors (GIST).
The new approval for indolent systemic mastocytosis was largely based on data from the PIONEER trial in which avapritinib showed significant improvements versus placebo in the primary (mean change in total symptom score) and all key secondary endpoints.
Avapritinib was well-tolerated. The most common adverse reactions were eye edema, dizziness, peripheral edema and flushing. Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1 percent of patients. Data from the PIONEER trial was recently presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting.
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