The FDA has approved a tyrosine kinase inhibitor to treat adults with an unresectable or metastatic gastrointestinal stromal tumor (GIST) that has a specific genetic mutation.

Avapritinib, marketed as Ayvakit, is for patients whose tumor harbors an exon 18 mutation in the PDGFRA gene, which encodes platelet–derived growth factor receptor α. The mutation is rare. According to the drug’s manufacturer, Blueprint Medicines Corporation in Cambridge, Massachusetts, about 6% of patients with a newly diagnosed GIST harbor this mutation. An estimated 4000 to 6000 US adults are diagnosed with a GIST annually.

Although the tyrosine kinase inhibitor imatinib is a recommended first-line treatment for advanced GIST, patients with the most commonPDGFRA exon 18 mutation, D842V, usually don’t respond to the therapy. Avapritinib’s indication includes this mutation. Michael Heinrich, MD, a professor of medicine at the Oregon Health & Science University and an investigator in the phase 1 trial that led to avapritinib’s approval, called the drug “a new standard of care.”

FDA已批准酪氨酸激酶抑制剂治疗患有不可切除或转移性胃肠道间质瘤（GIST）且具有特定基因突变的成年人。

市售Ayvakit的Avapritinib适用于肿瘤在PDGFRA基因中存在外显子18突变的患者，该基因编码血小板衍生的生长因子受体α。突变很少见。据该药物的生产商位于马萨诸塞州剑桥的Blueprint Medicines Corporation称，新诊断为GIST的患者中约有6％携带这种突变。估计每年有4000至6000名美国成年人被诊断出患有GIST。

尽管推荐酪氨酸激酶抑制剂伊马替尼是晚期GIST的一线治疗药物，但具有最常见PDGFRA外显子18突变D842V的患者通常对该疗法无反应。Avapritinib的适应症包括这种突变。俄勒冈健康与科学大学医学教授，第一阶段临床试验的研究者迈克尔·海因里希（Michael Heinrich）将该药物称为阿伐普利尼的批准，他将该药物称为“新的治疗标准”。