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

November 23, 2021

The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:

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 (≥30%) adverse reactions were stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common (≥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.

View full prescribing information for Fyarro.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’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 the 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 FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Follow the Oncology Center of Excellence on Twitter @FDAOncology

Please visit the FDA Hematology/Oncology (Cancer) Approvals & Safety Notifications webpage for a list of current and past approvals.