On November 22, 2021, the Food and Drug Administration approved sirolimus protein-bound particles for injectable suspension (albumin-bound) (Fyarro, Aadi Bioscience, Inc.) for adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Efficacy was evaluated in AMPECT (NCT02494570), a multicenter, single-arm clinical trial in 31 patients with locally advanced unresectable or metastatic malignant PEComa. Patients received sirolimus protein-bound particles at 100 mg/m2 on days 1 and 8 of each 21-day cycle until disease progression or unacceptable toxicity.
The main efficacy outcome measures were overall response rate (ORR) and duration of response (DOR), as assessed by blinded independent central review, using RECIST v.1.1. ORR was 39% (95% CI: 22%, 58%), including 2 patients with complete responses. Median DOR was not reached (95% CI: 6.5 months, not estimable). Among responders, 67% had a response lasting greater than 12 months and 58% had a response lasting greater than 24 months.
The most common (â‰¥30%) adverse reactions were stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common (â‰¥6%) grade 3 to 4 laboratory abnormalities were decreased lymphocytes, increased glucose, decreased potassium, decreased phosphate, decreased hemoglobin, and increased lipase.
The recommended dosage is 100 mg/m2 administered as an IV infusion over 30 minutes on days 1 and 8 of each 21-day cycle until disease progression or unacceptable toxicity.
This review used theÂ Assessment Aid, a voluntary submission from the applicant to facilitate the FDAâ€™s assessment.
This application was granted priority review, fast track designation, breakthrough therapy designation, and orphan drug designation. A description of FDA expedited programs is in theâ€¯Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDAâ€™sâ€¯MedWatch Reporting SystemÂ or by calling 1-800-FDA-1088.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCEâ€™sÂ Project FacilitateÂ at 240-402-0004 or emailÂ OncProjectFacilitate@fda.hhs.gov.
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