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The FDA the first treatment for adults with progressive desmoid tumors, a rare type of benign soft-tissue sarcoma.

The approval of oral nirogacestat (Ogsiveo) stipulates use in patients with progressive tumors that require systemic treatment. Historically, surgery has been standard of care for desmoid tumors, but tumors might not be amenable to surgery, and the condition has a high risk of recurrence -- factors that have stimulated investigation of systemic therapies. Nirogacestat is an inhibitor of gamma secretase, which modulates Notch pathway activation.

"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," said Mrinal Gounder, MD, of Memorial Sloan Kettering Cancer Center in New York City, in a from SpringWorks Therapeutics. "Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumor symptoms. ... This approval represents an important therapeutic advance for patients."

Desmoid tumors arise from . They often occur in the abdomen but also the shoulders, upper arms, and thighs. The tumors do not metastasize but are locally aggressive and can spread to tissues adjacent to the site of origin, often causing substantial pain. Complete surgical removal can be challenging in many cases.

Primary support for approval of nirogacestat came from the phase III randomized, placebo-controlled DeFi trial involving 142 adults with progressive desmoid tumors. The results showed that patients treated with nirogacestat had a 71% reduction in the risk of disease progression or death. At the time of the primary analysis, median progression-free survival had yet to be reached in the nirogacestat arm versus 15.1 months in the placebo arm.

Confirmed objective responses occurred in 41% of nirogacestat-treated patients and 8% of the placebo group. Median time to response was 5.6 months with nirogacestat as compared with 11.1 months with placebo. Patients allocated to nirogacestat also had significant improvement in pain, tumor-specific symptoms, physical/role functioning, and overall health-related quality of life.

The most common side effects observed with nirogacestat (≥15% of patients) were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.

"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 tumors," said Richard Pazdur, MD, of the FDA's Oncology Center of Excellence, in a statement. "Desmoid tumors are rare tumors that can lead to severe pain and disability. Today's approval will offer the first approved treatment option for patients beyond surgery and radiation."

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