June 19, 2023
PITTSBURGH – Blueprint Medicines recently announced the approval of AYVAKIT® (avapritinib) for the expanded indication of adults with Indolent Systemic Mastocytosis (ISM). AYVAKIT was previously approved for the treatment of adults with Advanced Systemic Mastocytosis (SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL), as well as for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. PANTHERx Rare Pharmacy dispenses AYVAKIT for all previously approved indications and will now dispense AYVAKIT for the ISM patient population.
SM is a rare disease that is driven by the KIT D816V mutation in about 95% of cases. This mutation causes the uncontrolled growth and activation of mast cells, which in turn results in chronic, often unpredictable, and potentially severe symptoms across many different organ systems. The vast majority of people living with SM are diagnosed with ISM. People living with ISM may experience a broad range of symptoms. Common symptoms include anaphylaxis, rash, itching, diarrhea, brain fog, fatigue, and bone pain, and these symptoms often persist despite treatment with multiple therapies aimed at improving symptoms. These symptoms can have profound effects on people’s lives and can interfere with their ability to work and complete their normal daily activities, and may even lead to patients isolating themselves to protect against possible unexpected triggers.
AYVAKIT is a kinase inhibitor designed to target the KIT D816V mutation, the primary driver of the disease. In the registrational clinical trial for ISM, treatment with AYVAKIT significantly reduced mast cell burden and improved patient symptoms. The most common adverse reactions for AYVAKIT (≥10 percent) were eye edema, dizziness, peripheral edema and flushing.
About PANTHERx Rare
PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.
For more information, please contact marketing@pantherxrare.com.
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