Aadi clinches first FDA nod for ultrarare sarcoma drug Fyarro

Aadi clinches first FDA nod for ultrarare sarcoma drug Fyarro

Aadi Bioscience’s new cancer med Fyarro boasts two firsts: It’s the California-based company’s first commercial product, and it’s the first treatment to take aim at an ultrarare and aggressive form of sarcoma.

The FDA on Tuesday approved Fyarro, also known as sirolimus protein-bound particles for injectable suspension, in adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

With an approval in hand, Aadi says it plans to launch the drug in the first quarter of 2022. It’s set a wholesale acquisition cost of about $39,000 a month, or $468,000 a year, a company spokesperson said over email. 

Malignant PEComas are a family of ultrarare tumors that crop up in fewer than 1 in 1,000,000 people globally each year, the spokesperson added. They’re a type of sarcoma that form in soft tissues like the stomach, intestine, lungs and female reproductive organs along with genitourinary organs such as the kidneys and bladder. 

RELATED: Ready to rival J&J, Takeda’s growth plan bears fruit with FDA nod for niche lung cancer drug Exkivity

Prior to Fyarro’s approval, there were no treatments specifically designed for PEComa patients, the Aadi spokesperson noted. Those patients’ prognoses are often poor, too. After chemotherapy, patients typically survive about 16 months, the spokesperson explained. Meanwhile, nearly three-fourths of PEComa patients will develop metastatic disease, most often in the lungs or liver, within one year of their diagnosis, he added. 

The FDA gave Fyarro the thumbs-up based on data from a phase 2 registrational trial. Thirty-nine percent of patients who received Fyarro in Aadi’s AMPECT study achieved an overall response, with two patients hitting a complete response after prolonged follow-up, the company said. Among that group of responders, 92% had a response lasting six months or more. Sixty-seven percent had a response lasting a year or more, while 58% had a response lasting two or more years, Aadi said. 

To prepare for the launch, the company has assembled a “strong” sales team and laid out a “thoughtful” strategy for its market debut, Brendan Delaney, chief operating officer at Aadi, said in a release. 

RELATED: ChemoCentryx scores first FDA approval in 24-year history, and CEO Schall sees a blockbuster in the making

“With Fyarro’s demonstrated clinical profile we believe it will become a standard of care for advanced malignant PEComa,” he added. 

Meanwhile, Aadi’s top brass aren’t the only ones trumpeting Fyarro’s potential popularity. The drug is bound to be “welcomed” by doctors as the only approved therapeutic for patients with advanced malignant PEComa, Andrew Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute and the principal investigator in Fyarro’s AMPECT trial, said in a statement.

This story is brought to you by Fierce Pharma.

Leave a Reply

Your email address will not be published. Required fields are marked *