The Food and Drug Administration (FDA) has approved Fyarro™ (sirolimus protein-bound particles [albumin-bound]) for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Fyarro is an mTOR inhibitor bound to human albumin. The approval was based on data from the multicenter phase 2 AMPECT study (ClinicalTrials.gov Identifier: NCT02494570), which evaluated the efficacy of Fyarro in 34 adults aged 18 years and older with advanced malignant PEComa. Patients received Fyarro intravenously weekly until disease progression or unacceptable toxicity. The primary endpoint was overall response rate (ORR).
Findings showed that among 31 evaluable patients, the confirmed ORR was 39% (n=12/31; 95% CI, 22-58), with 2 patients achieving a complete response after long-term follow-up. After a median follow-up of 36 months, the median duration of response was not yet reached and ranged from 5.6 to greater than 55.5 months. Among responders, 92% of patients had a response lasting at least 6 months, 67% had a response lasting at least 12 months, and 58% had a response lasting at least 2 years.
“In our AMPECT trial, Fyarro demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, with an acceptable and manageable safety profile,” said Andrew Wagner, MD, PhD, a senior oncologist at Dana-Farber Cancer Institute and the principal investigator in the pivotal AMPECT registrational trial. “This is a drug that will be welcomed by the physician community as the only approved therapeutic option for patients with advanced malignant PEComa.”
Fyarro is contraindicated in patients with a history of severe hypersensitivity to sirolimus, other rapamycin derivatives, or albumin. Treatment has also been associated with stomatitis, myelosuppression, infections, hypokalemia, hyperglycemia, interstitial lung disease, hemorrhage, and hypersensitivity reactions.
The most common adverse reactions (more than 30%) reported with Fyarro include stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia.
Fyarro is expected to be available in the first quarter of 2022.
Aadi Bioscience announces FDA approval of its first product Fyarro™ for patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). News release. Aadi Bioscience. Accessed November 23, 2021. https://www.globenewswire.com/news-release/2021/11/23/2339619/0/en/Aadi-Bioscience-Announces-FDA-Approval-of-its-First-Product-FYARRO-for-Patients-with-Locally-Advanced-Unresectable-or-Metastatic-Malignant-Perivascular-Epithelioid-Cell-Tumor-PECom.html.