Aadi Bioscience announced this week that the FDA has given the green light to its treatment for a very rare cancer with a low chance of survival.
According to the company, the agency has approved its intravenous drug called Fyarro, which was developed to treat locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). It is the first time the agency has given the OK to a drug for the indication.
Instances of malignant PEComas, which are sarcomas that form in the soft tissues of the body, are rare — the condition impacts about 100-300 new patients in the U.S. each year. But for the patients who receive the diagnosis, the prognosis is dim. After receiving chemo, the standard of care, the median rate of survival is about 16 months.
According to Aadi, a phase 2 trial of Fyarro showed an overall response rate of 39%, with two patients achieving a complete response after a prolonged follow up.
“Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to 2 years,” the company said in a statement.
The company said that the drug, which received priority review from the FDA, will likely now become the standard of care for malignant PEComa.
