FDA approves first therapy for rare non-cancerous tumours

FDA approval

SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has become the first drug to be approved for desmoid tumours in the US, following the go-ahead from the US Food and Drug Administration (FDA).

Desmoid tumours are non-cancerous but can be locally aggressive. Although surgical removal has historically been the treatment of choice, there is a high risk that the tumour will return or that other health challenges will occur after removal.

“The FDA continues to address unmet medical need and advance the development of safe and effective therapies for the millions of Americans whose lives are affected by rare tumours,” said Richard Pazdur, Director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Desmoid tumours are rare tumours that can lead to severe pain and disability. Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

Results from the Phase III DeFi trial demonstrated that Ogsiveo provided clinically meaningful and statistically significant improvement in progression-free survival compared to placebo. The objective response rate was also statistically different between the two arms, with a response rate of 41% in the Ogsiveo arm and 8% in the placebo arm.

“Desmoid tumors can have a significant impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence. This approval represents an important therapeutic advance for patients,” said Mrinal M Gounder, sarcoma medical oncologist at Memorial Sloan Kettering Cancer Center (MSK) in New York City and an investigator in the DeFi trial.

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