The U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is the first and only FDA-approved treatment for advanced malignant PEComa in adults.
About Malignant PEComa
Advanced malignant PEComa, defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,’ are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract with a female predominance) and can have an aggressive clinical course including distant metastases and ultimately death. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.
FYARRO is an mTOR inhibitor indicated for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Understanding DNA Damage Response or DDR and Cancer Treatment
What is DNA Damage Response or DDR?
In the Phase 2 clinical trial leading to FDA approval the overall response rate to Fyarro was 39% with 2 patients achieving a complete response. The median duration of response was not reached with a median follow-up of 36 months. Among patients responding to treatment, 92% had a response lasting greater than or equal to 6 months and 58% had a response lasting greater than or equal to 2 years.
Side effects associated with FYARRO include mouth sores, low blood counts, rash, fatigue and infection.
The approval of FYARRO provides a new therapeutic option to offer malignant PEComa patients."