FDA Approves First Treatment and Drug Specifically for PEComa
The FDA has approved the first treatment specifically for patients with an ultra-rare and aggressive form of cancer seen mostly in women.
Made by Aadi Bioscience and called Fyarro, the treatment, sirolimus protein-bound particles for injectable suspension, is aimed at people with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
The approval was based on a phase 2 trial of 31 patients in which Fyarro showed an overall response rate of 39 percent, with two patients achieving a complete response after prolonged followup, Aadi said.
Among patients who responded to the drug, 92 percent had a response lasting greater than or equal to six months, while 67 percent had a response lasting longer than or equal to 12 months, and 58 percent had a response lasting greater than or equal to two years, the company said.
Aadi said it’s planning Fyarro’s launch in the first quarter of 2022.