On January 17, 2020 PANTHERx Rare Pharmacy reported that it has been selected by Blueprint Medicines Corporation as a very limited distribution partner for breakthrough drug AYVAKIT (avapritinib) (Press release, Blueprint Medicines, JAN 17, 2020, View Source [SID1234553314]). It has been approved for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. With nationwide distribution of AYVAKIT, PANTHERx establishes a formidable presence in the precision oncology space.

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"PANTHERx is thrilled to be chosen by Blueprint Medicines to collectively make historic strides in precision oncology," said Dr. Gordon Vanscoy, Chairman and CEO of PANTHERx. "As a partner, we are grateful to offer hope to a genomically defined population of patients with rare gastrointestinal stromal tumors. PANTHERx is proud to be part of an elite network advancing precision oncology and bringing new hope to rare cancer patients."

AYVAKIT is a potent and selective KIT and PDGFRA inhibitor. It is the only FDA-approved type 1 inhibitor for GIST that works by directly binding to the active kinase conformation from which mutant KIT and PDGFRA signal. AYVAKIT has demonstrated inhibition of a broad range of KIT and PDGFRA mutations associated with GIST, including potent clinical activity against activation loop mutations that are associated with resistance to currently approved therapies.

The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation and one for the treatment of advanced systemic mastocytosis (SM), including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

For more information about PANTHERx RxARECARE services for patients with GIST, contact us at 833-918-2015.

Gastrointestinal Stromal Tumors

Gastrointestinal Stromal Tumors (GISTs) are a rare form of cancer located in the gastrointestinal (GI) tract. More than half of GISTs start in the stomach although many start in the small intestine and other parts of the GI tract. Doctors will look at specific features of a GIST to help predict whether it is likely to grow and spread to other parts of the body. GISTs are evaluated based on the size of the tumor, where it is located in the GI tract, and how fast the tumor cells are dividing (mitotic rate).

The GIST tumors often grow into empty spaces inside the GI tract so they may not cause symptoms until they reach a certain size or are in a certain location. As a result, a small tumor may not cause any symptoms. Common symptoms of GISTs include: include throwing up bright red or dark blood, bleeding into the stool, abdominal pain, a noticeable mass or swelling in the abdomen, nausea or vomiting, loss of appetite and weight loss, and feeling full after eating small amounts of food.

Treatment options depend on a several factors, including the tumor size, the mitotic rate, where the tumor is located in the GI tract, the genetic makeup of the tumor, and if the tumor has spread to other parts of the body. Treatment options include surgery to remove the tumor and targeted therapies with tyrosine kinase inhibitors (TKIs).

For more information about the symptoms, diagnosis, and treatment of Gastrointestinal Stromal Tumors please click here (View Source).

About PANTHERx Rare Pharmacy

PANTHERx Rare Pharmacy, the nation’s only health entity with an Accredited Distinction (ACHC) in Rare Pharmacy, is headquartered in Pittsburgh, Pennsylvania. PANTHERx Rare’s mission is to transform lives by delivering medical breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Every day we Define RxARE, by developing focused solutions, bold innovations, and novel clinical services associated with the life-transforming therapies that we deliver.

PANTHERx was awarded 2018 Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy. Zitter Health Insights awarded PANTHERx the Specialty Pharmacy Patient Choice Award winner for two consecutive years. As a quad-accredited pharmacy, PANTHERx holds accreditations from URAC, VIPPS, ACHC, and CPPA.