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Ayvakit based on Avapritinib as the first treatment for indolent systemic mastocytosis. FDA approved


The U.S. Food and Drug Administration ( FDA ) has approved Ayvakit ( Avapritinib ) for the treatment of adults with indolent systemic mastocytosis ( ISM ).

The approval of Ayvakit in indolent systemic mastocytosis is based on data from the double-blind, placebo-controlled PIONEER trial in which patients received Ayvakit 25 mg once daily plus best supportive care ( Ayvakit ) or placebo plus best supportive care ( placebo ).
Ayvakit has demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

Ayvakit was well-tolerated with a favorable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity.
The most common adverse reactions for Ayvakit ( greater than or equal to 10% ) were eye edema, dizziness, peripheral edema and flushing.
Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1% of patients.

Ayvakit is a precision therapy approved by the FDA for the treatment of three indications: adults with indolent systemic mastocytosis, adults with advanced systemic mastocytosis, including aggressive systemic mastocytosis ( ASM ), systemic mastocytosis with an associated hematological neoplasm ( SM-AHN ) and mast cell leukemia ( MCL ), and adults with unresectable or metastatic gastrointestinal stromal tumor ( GIST ) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Systemic mastocytosis is a rare disease driven by the KIT D816V mutation in about 95% of cases.
Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems.
The vast majority of those affected have indolent systemic mastocytosis.
A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with indolent systemic mastocytosis despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers.
A minority of patients have advanced systemic mastocytosis, which encompasses a group of high-risk systemic mastocytosis subtypes including aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm and mast cell leukemia.
In addition to mast cell activation symptoms, advanced systemic mastocytosis is associated with organ damage due to mast cell infiltration and poor survival. ( Xagena )

Source: Blueprint Medicines, 2023

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