First pill for desmoid tumours approved

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The US Food and Drug Administration (FDA) has approved the first drug for desmoid tumours (desmoid fibromatosis), an oral gamma secretase inhibitor. SpringWorks Therapeutics’ Ogsiveo (nirogacestat) tablets are authorised for adults with progressing desmoid tumours who require systemic treatment.

A new treatment for desmoid tumours

As a subtype of soft tissue sarcomas: “Desmoid tumours are rare [non-cancerous] tumours that can lead to severe pain and disability… [Ogsiveo is] the first approved treatment option for patients beyond surgery and radiation,” noted Dr 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.

The FDA’s approval of Ogsiveo is based on results from the Phase III DeFi clinical trial, which were published this year in the New England Journal of Medicine.

According to the FDA, this clinical trial evaluated 142 adult patients with progressing desmoid tumours not suitable to surgery. Patients were randomised to receive 150mg of Ogsiveo or placebo.

Phase III trial results for Ogsiveo

Data showed that Ogsiveo improved progression-free survival (PFS), demonstrating a statistically significant improvement over placebo with a 71 percent reduction in the risk of progression of desmoid tumours. Additionally, objective response rate was also statistically different between the two arms. A response rate of 41 percent was observed in the Ogsiveo arm, while this rate was eight percent in the placebo arm.

“We are pleased with the broad label, which includes all progressing adult patients and specifically references improvement in pain, and believe Ogsiveo has the potential to become the new standard of care for people living with these devastating tumours,” Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics commented.

SpringWorks Therapeutics stated that it expects to file a Marketing Authorisation Application (MAA) for Ogsiveo in desmoid tumours with the European Medicines Agency (EMA) in the first half of 2024.