FDA approves costly new treatment for ultra-rare, aggressive form of sarcoma – Endpoints News
The FDA on Tuesday approved Aadi Bioscience’s first drug and the first treatment approved specifically for patients with an ultra-rare and aggressive form of sarcoma that occurs primarily in women.
The approval of the drug, known as Fyarro, is for people with locally advanced, unresectable or metastatic malignant perivascular epithelioid tumor (PEComa), and is based on a Phase II trial.
The results showed an overall response rate assessed by independent review of 39% (12/31), with two patients achieving a complete response after prolonged follow-up, Aadi said. The company also said that among the respondents, 92% had a response of six months or more; 67% had a duration of response greater than or equal to 12 months; and 58% had a response lasting more than or equal to two years.
The company said the price of the drug would be around $ 39,000 per month, or $ 468,000 per year.
Aadi COO Brendan Delaney said during Tuesday’s investor call: âThe wholesale acquisition cost of FYARRO for a 100 milligram single-use vial is $ 6,785.00, taking into account the variability of patient body surface area and dose changes, we project the wholesale acquisition cost for one month of FYARRO. therapy at about $ 39,000. Legal government rebates such as Medicaid rebates, sales to 340 billion covered entities and VA facilities, among others, will reduce the net price Aadi Bioscience receives by about 15-20%.
Andrew Wagner, principal oncologist at the Dana-Farber Cancer Institute and principal investigator of the pivotal trial, said in a statement that Fyarro would provide doctors with “a new weapon to treat patients with this rare disease.”
The company’s shares rose more than 25% in pre-market trading on Tuesday.
Fyarro was created by Aadi Founder and CEO Neil Desai, the former global head of R&D at Abraxis, who attempted to develop an approach similar to the one he and Patrick Soon-Shiong used to create the drug. successful cancer drug Abraxane, which is now in short supply. Abraxane was decades-old chemotherapy encased in a layer of albumin that prompts tumor cells to absorb the tumor-killing agent.
Along with Fyarro, Desai took a decades-old immunomodulatory drug, rapamycin, and coated it with a similar layer of albumin. Once absorbed by the tumor, it breaks down mTOR, a protein that has long been linked to tumor proliferation, among others.
The company did not respond to a request for comment on the price of the new treatment.
In May, Aadi merged with struggling public biotech Aerpio Pharmaceuticals and raised $ 155 million from private investors to market Fyarro.