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FDA Approves Sirolimus Protein-Bound for Perivascular Epithelioid Cell Tumors

Renal angiomyolipoma.

The FDA has approved sirolimus protein-bound particles for injectable suspension (albumin-bound) (Fyarro™, Aadi Bioscience) for patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.

"Malignant perivascular epithelioid cell tumor is a rare, aggressive sarcoma," wrote Andrew J. Wagner, MD, PhD, Medical Director of Ambulatory Oncology at Dana-Farber Cancer Institute and Associate Professor of Medicine at Harvard Medical School, and colleagues, in their publication of the AMPECT trial (NCT02494570), on which the approval was based. "Cytotoxic chemotherapies have limited benefit for patients with advanced disease."

The multicenter, single-arm clinical trial enrolled 31 patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor. Patients received 100 mg/m2 sirolimus protein-bound particles on Day 1 and Day 8 of each 21-day cycle until disease progression or unacceptable toxicity. The primary end point was overall response rate; a key secondary end point was duration of response.

Sirolimus protein-bound produced overall response rate was 39%, which included 2 patients who achieved complete responses; the median duration of response was not reached. Among the patients who responded, 67% had a response lasting longer than 12 months, and 58% had a response lasting longer than 24 months.

The most common adverse reactions occurring in at least 30% of patients included stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia. The most common grade 3 or 4 laboratory abnormalities occurring in at least 6% of patients included decreased lymphocytes, increased glucose, decreased potassium, decreased phosphate, decreased hemoglobin, and increased lipase.

"The high disease control rate with manageable toxicities suggest that sirolimus [protein-bound] is effective and represents an important new treatment option for these patients," concluded Dr. Wagner and colleagues in their abstract presented at the 2020 American Society of Clinical Oncology (ASCO) Annual Meeting and published in the Journal of Clinical Oncology.

The recommended dose of sirolimus protein-bound particles is 100 mg/m2 via intravenous infusion over 30 minutes on Day 1 and Day 8 of each 21-day cycle until disease progression or unacceptable toxicity.

For More Information

Wagner AJ, Ravi V, Riedel RF, et al (2020). Long-term follow-up for duration of response (DoR) after weekly nab-sirolimus in patients with advanced malignant perivascular epithelioid cell tumors (PEComa): results from a registrational open-label phase II trial, AMPECT. J Clin Oncol (ASCO Annual Meeting Abstracts), 38(suppl_15). Abstract 11516. DOI:10.1200/JCO.2020.38.15_suppl.11516


Clinicaltrials.gov (2021). A phase 2 study of ABI-009 in patients with advanced malignant PEComa (AMPECT). NLM identifier: NCT02494570.

Fyarro™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) prescribing information (2021). Aadi Bioscience. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/213312lbl.pdf

US Food & Drug Administration (2021). FDA approves sirolimus protein-bound particles for malignant perivascular epithelioid cell tumor. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-sirolimus-protein-bound-particles-malignant-perivascular-epithelioid-cell-tumor

Image credit: KGH. Licensed under CC BY-SA 3.0


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