NEW YORK – Aadi Bioscience said on Monday that the US Food and Drug Administration has accepted its new drug application seeking approval of the mTOR inhibitor nab-sirolimus (Fyarro) as a treatment for advanced malignant PEComa, a rare sarcoma often characterized by TSC1 or TSC2 gene mutations.
The FDA also granted priority review for this nab-sirolimus indication and set a target action date of Nov. 26. Previously, nab-sirolimus was granted orphan drug, fast-track, and breakthrough therapy designations for this indication.
Aadi submitted data from the Phase II AMPECT registrational trial. That study evaluated nab-sirolimus in 34 patients with advanced, malignant PEComa and achieved a 39 percent overall response rate.
Based on discussions with the FDA, Aadi also said it plans to begin a tumor-agnostic registrational trial of nab-sirolimus by the end of 2021 in mTOR inhibitor-naive solid tumors harboring TSC1 or TSC2 inactivating alterations. The company is also planning to study nab-sirolimus in combination with other drugs.
Earlier this year, Los Angeles-based Aadi merged with Aerpio Pharmaceuticals, planning to raise $155 million in a private investment in public equity financing round to advance nab-sirolimus.