FDA approves new therapy to treat a rare mutation in patients with gastrointestinal stromal tumors

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FDA approves new therapy to treat a rare mutation in patients with gastrointestinal stromal tumors

Today, the U.S. Food and Drug Administration approved Ayvakit (avapritinib) for the treatment of adults with unresectable (unable to be removed with surgery) or metastatic (when cancer cells spread to other parts of the body) gastrointestinal stromal tumor (GIST) – a type of tumor that occurs in the gastrointestinal tract, most commonly in the stomach or small intestine – harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. This approval includes GIST that harbors a PDGFRA D842V mutation, which is the most common exon 18 mutation. Ayvakit is a kinase inhibitor, meaning it blocks a type of enzyme called a kinase and helps keeps the cancer cells from growing. Common side effects for patients taking Ayvakit were edema (swelling), nausea, fatigue/asthenia (abnormal physical weakness or lack of energy), cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation (secretion of tears), abdominal pain, constipation, rash and dizziness. Ayvakit can cause intracranial hemorrhage (bleeding that occurs inside the skull) in which case the dose should be reduced, or the drug should be discontinued. Ayvakit can also cause central nervous system effects including cognitive impairment, dizziness, sleep disorders, mood disorders, speech disorders and hallucinations. If this happens, depending on the severity, Ayvakit should be withheld and then resumed at the same or reduced dose upon improvement or permanently discontinued. Health care professionals should advise pregnant women that Ayvakit may cause harm to a developing fetus or newborn baby. Additionally, the FDA advises health care professionals to tell females of reproductive potential, and males with female partners of reproductive potential, to use effective contraception during treatment with Ayvakit and for six weeks after the final dose.

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