FDA Approves Sirolimus Protein-Bound Particles for Malignant Perivascular Epithelioid Cell Tumor

November 23, 2021

On November 22, 2021, the U.S. Food and Drug Administration (FDA) approved (https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-sirolimus-protein-bound-particles-malignant-perivascular-epithelioid-cell-tumor) sirolimus protein-bound particles for injectable suspension (Fyarro™) for adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

FDA Approves Sirolimus Protein-Bound Particles for Malignant Perivascular Epithelioid Cell Tumor

Efficacy was evaluated in a multicenter, single-arm clinical trial (AMPECT; NCT02494570) 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 they experienced disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate (ORR) and duration of response (DOR), as assessed by a blinded independent central review using response evaluation criteria in solid tumors 1.1. ORR was 39% (95% CI = 22%, 58%), including two 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 adverse reactions reported in 30% or more of patients were stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common grade 3–4 laboratory abnormalities reported in 6% or more of patients were decreased lymphocytes, potassium, phosphate, and hemoglobin and increased lipase and glucose.

The recommended dosage is 100 mg/m2 administered via IV infusion over 30 minutes on days 1 and 8 of each 21-day cycle until patients experience disease progression or unacceptable toxicity.

View the full prescribing information for sirolimus protein-bound particles (https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213312lbl.pdf).

The review used the Assessment Aid (https://www.fda.gov/about-fda/oncology-center-excellence/assessment-aid), a voluntary submission from the applicant to facilitate FDA’s assessment.

FDA granted the application priority review, fast track designation, breakthrough therapy designation, and orphan drug designation. A description of FDA expedited programs is in the Guidance for IndustryꟷExpedited Programs for Serious ConditionsꟷDrugs and Biologics (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics). 

Healthcare professionals should report all serious adverse events they suspect are associated with the use of any medicine and device to FDA’s MedWatch Reporting System (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 800-FDA-1088. 


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