Brendan Delaney, MBA, Chief Operating Officer of Aadi Bioscience, discusses albumin-bound sirolimus (Fyarro), the recently-approved treatment for malignant perivascular epithelioid cell tumor (PEComa).
PEComa is an ultra-rare family of sarcomas. Most PEComa begin as benign tumors which can be removed surgically. However, when these tumors are malignant, they become aggressive. Often it is diagnosed as a painful mass in the abdominal region. When it is possible, surgery is usually the preferred treatment for PEComa; however, due to the aggressive nature of these tumors when malignant, chemotherapy has historically been used. Unfortunately, chemotherapy has been shown in the literature to have minimal benefit for patients with PEComa.
Recently, Aadi Bioscience received FDA approval for albumin-bound sirolimus for the treatment of adult patients with locally advanced unresectable or metastatic malignant PEComa.
The approval of the drug was largely based on positive results from the phase 2 AMPECT trial. The overall response rate in this trial, as assessed by independent review, was 39% (12/31), with 2 patients achieving a complete response after prolonged follow up. The median duration of response has not been reached with a median follow-up of 36 months. Among responders, 92% had a response lasting over 6 months; 67% had a response lasting less than 12 months; and 58% had a response lasting over 2 years. Grade 3 non-hematologic events occurring in more than 10% of patients included stomatitis, rash, fatigue and infections. Grade 3 laboratory abnormalities occurring in more than 10% of patients that worsened from baseline included lymphocytopenia, increased glucose, and decreased potassium.
To learn more about PEComa and other rare cancers, visit checkrare.com/diseases/cancers/