FDA approves pricey new treatment for ultra-rare and aggressive form of sarcoma
The FDA on Tuesday approved Aadi Bioscience’s first drug and the first treatment approved specifically for patients with an ultra-rare and aggressive form of sarcoma that occurs mostly in women.
The approval of the drug, known as Fyarro, is for those with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa), and is based on a Phase II trial.
Results showed an overall response rate as assessed by independent review of 39% (12/31), with two patients achieving a complete response after prolonged follow up, Aadi said. The company also said that among responders, 92% had a response lasting greater than or equal to six months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to two years.
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