FDA Approves Therapy for Gastrointestinal Stromal Tumors

January 13, 2020

The approval comes after the results of a clinical trial that showed 61% of patients had a response lasting six months or longer.

On Jan. 9, 2020, FDA announced the approval of Blueprint Medicines’ Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation or PDGFRA D842V mutation.

According to an FDA press release, the approval comes after the results of a clinical trial involving 43 patients with GIST harboring a PDGFRA exon 18 mutation, including 38 participants with the PDGFRA D842V mutation, that measured complete or partial shrinkage of their tumors during treatment. Each patient received 300 mg or 400 mg of the drug once daily and 61% of patients had a response lasting six months or longer.

“GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harboring this mutation,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in FDA’s Center for Drug Evaluation and Research, in the press release. “Clinical trials showed a high response rate with almost 85% of patients experiencing tumor shrinkage with this targeted drug.”

Source: FDA