First approval for U.S. rare sarcoma treatment

[의학신문·일간보사=김자연 기자] A rare sarcoma, perivascular epithelial cell tumor (PEComa), was approved for the first time in the United States.

FDA approved Adi Bio’s Fyarro (sirolimus protein-bound particles) for the treatment of locally advanced unresectable and metastatic malignant PEComa.

This is an albumin-binding mTOR inhibitor intravenous injection, and as a result of the phase 2 clinical trial, 39% of 31 patients responded, 2 of them achieved a complete response, 67% of responders lasted for more than 1 year, and 58% of them lasted for more than 2 years.

As with other mTOR series, the prescribing information includes stomatitis, myelosuppression, infection, hypokalemia, hyperglycemia, interstitial lung disease, bleeding, and hypersensitivity reactions as cautions and warnings.

In response, Adi Bio expects it to be a standard treatment, and the wholesale acquisition price for a single 100mg vial in the United States is $6785, which is about $39,000 for one month of treatment.