(PEComa) FDA Approves Fyarro for Treatment
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This article is a summary of international medical information and is not medical advice; it cannot replace the diagnosis or treatment plan of your attending physician. The medical technologies, drug information and clinical data presented here are compiled from public literature and official statements of major Japanese medical institutions; the applicability and outcome of any therapy vary with each patient and must be assessed individually by a qualified physician.
On November 22, 2021, the U.S. Food and Drug Administration (FDA) approved Fyarro (sirolimus protein-bound particles) for the treatment of locally advanced unresectable and metastatic malignant perivascular epithelioid cell tumors (PEComa).
This multicenter trial enrolled patients with locally advanced unresectable and metastatic malignant PEComa (N=31), who received Fyarro (100 mg/m² on Days 1 and 8 of each 21-day cycle) until disease progression or intolerable adverse events.
The primary endpoint objective response rate (ORR) was 39%, including 2 patients with complete response. The median duration of response was not reached; however, 67% of responding patients had responses lasting more than 12 months, and 58% had responses lasting more than 24 months.
The most common adverse events (≥30%) were mouth sores, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, weight loss, loss of appetite, cough, vomiting, and taste disturbance. Grade 3–4 adverse events occurring in ≥6% of patients included lymphocytopenia, hyperglycemia, hypokalemia, hypophosphatemia, decreased hemoglobin, and elevated lipase.
The recommended dose of Fyarro is 100 mg/m² on Days 1 and 8 of a 21-day cycle, administered until disease progression or intolerable adverse events.
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