On November 22, 2021, the FDA approved Fyarro (sirolimus protein-bound particles for injectable suspension (albumin-bound); from Aadi Bioscience) for adults with locally advanced, unresectable or metastatic malignant perivascular epithelioid-cell tumor (PEComa), a rare type of mesenchymal tumors that can occur in any part of the body.
This FDA approval was based on a multicenter, single-arm clinical trial of 31 patients with locally advanced, unresectable or metastatic malignant PEComa. Patients received Fyarro on days 1 and 8 of each 21-day cycle, until disease progression or unacceptable side effects.
The overall response rate was 39%, which included 2 patients with complete responses. The average duration of response was not reached (meaning patients were still responding at the time of the analysis). Of the 39% of patients who had a response, 67% had a response lasting more than 12 months, and 58% had a response lasting more than 24 months.
The most common side effects were stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common serious (grade 3 or 4) laboratory abnormalities were decreased lymphocytes, increased glucose, decreased potassium, decreased phosphate, decreased hemoglobin, and increased lipase levels.
