FDA’s Acceptance of AYVAKIT’s New Drug Application Offers Hope to Metastatic GIST Patients


Surgery has been found to often be the best option towards a cure for a cancerous tumor (resectable) but there are many other tumors that are diagnosed as unresectable (cannot be cured with surgery). A metastatic gastrointestinal stromal tumor (GIST) fits solidly into that category.

Blueprint Medications’ recent announcement via its press release offers hope to metastatic GIST patients through the FDA’s approval of its drug AYVAKIT™ (avapritinib).

The approval came about as a result of positive results from the NAVIGATOR Phase I trial. Approximately eighty-five of the participants experienced tumor shrinkage.

Favorable safety results from several other trials also contributed to the FDA’s decision.

Precision Therapy and Precision Medicine

The drug falls under the category of a precision therapy. It will be used to treat patients who were diagnosed by a doctor’s observation of changes in the patient’s tumor.


GIST is categorized as a sarcoma which is a rare form of cancer that affects bone or connective tissue of the gastrointestinal tract.

The walls of the gastrointestinal tract contain specialized nerve cells that control digestive functions and move food through the intestinal tract. GISTs are formed by DNA mutations in one or more of these cells.

Over fifty percent of GISTs begin in the stomach. Others may begin somewhere in the small intestine or in any part of the gastrointestinal system.

AYVAKIT™ was developed as a treatment for GIST with platelet-derived growth factor receptor-alpha (PDGFRA) exon 18 mutations.

Approximately ten percent of reported cases of GIST were linked to mutations in the PDGFRA gene. To date, there has been no effective therapy for GIST with the PDGFRA mutations.

About Blueprint Medicines

Blueprint Medicines is a precision therapy company with a focus on genomically defined cancers, immunotherapy and rare diseases.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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