Today, the U.S. Food and Drug Administration (FDA) has approved Ogsiveo (nirogacestat) tablets as the first therapy designed for adults with progressing desmoid tumors, a rare subtype of soft tissue sarcomas. Desmoid tumors, though non-cancerous, are locally aggressive, causing pain and impaired mobility.
Traditionally, surgery was the go-to option, but with a high risk of recurrence, systemic therapies have gained attention. Ogsiveo's approval marks a pivotal moment for those with these challenging tumors.
Richard Pazdur, M.D. 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, emphasized the FDA's commitment to advancing safe and effective therapies, stating, “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.”
The efficacy of Ogsiveo was assessed in a clinical trial involving 142 patients with progressing desmoid tumors, where Ogsiveo demonstrated significant improvements in progression-free survival and tumor shrinkage.
Administered orally at 150mg twice daily, Ogsiveo outperformed the placebo, with a remarkable 41% positive response rate compared to 8%. Patient-reported assessments also favored Ogsiveo.
Common side effects observed in at least 15% of the patients included diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Ogsiveo's expedited approval, coupled with FDA designations like Priority Review, Fast Track, and Breakthrough Therapy, underscores its potential to significantly improve desmoid tumor treatment. The Orphan-Drug designation further incentivizes its development for rare diseases.
The approval, granted to SpringWorks Therapeutics Inc., signifies a crucial step forward, providing hope and improved outcomes for desmoid tumor patients.
If you wish to find out more, please read FDA's official announcement.