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Aadi Bioscience recently launched Fyarro, the first FDA-approved therapy to treat an ultra-rare sarcoma.
PANTHERx Rare has been selected by Aadi Bioscience as the exclusive U.S. pharmacy distribution partner for new drug Fyarro (sirolimus protein-bound particles). Fyarro, an albumin-bound mTOR inhibitor administered as an injectable suspension, is the first FDA-approved therapy for the treatment of advanced malignant perivascular epithelioid cell neoplasm (PEComa), an ultra-rare sarcoma.
The FDA approved Fyarro in November 2021, and Aadi Bioscience launched the therapy in February 2022. Fyarro was also recently added to the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology as the only preferred treatment regimen for malignant PEComa.
PANTHERx Rare is a pharmacy in the United States focused on rare diseases. It recently earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care. PANTHERx, headquartered in Pittsburgh, Pa., is licensed in all 50 states, and also holds accreditations from URAC and National Association of Boards of Pharmacy.
Aadi Bioscience has also launched Aadi Assist, a patient support program that offers resources for patients, including information about co-pay assistance, referrals, educational resources, and verification of benefits.
“We are committed to working with payers and healthcare providers across the country to help ensure access to Fyarro,” Brendan Delaney, chief operating officer of Aadi Bioscience, said in a press release.
Advanced PEComa is a rare, aggressive subset of soft tissue sarcomas with an undefined cell of origin. Malignant PEComas can impact almost any body site, but it typically is found in the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract. It can be aggressive, leading to metastases.
In the phase 2 registrational AMPECT trial, Fyarro demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa. The overall response rate as assessed by independent review was 39% (12/31). The median duration of response has not been reached with a median follow-up of 36 months, and a range of 5.6 to 55.5+ months and ongoing. Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to two years.
Aadi Bioscience continues to study Fyarro in other tumor types with alterations in mTOR pathway genes. The PRECISION-1 study, a registrational trial in patients with solid tumors with pathogenic alterations in TSC1 or TSC2 genes, is now open for enrollment.