Ogsiveo (nirogacestat) tablets have received approval from the Food and Drug Administration for use in adult patients with progressing desmoid tumors requiring systemic treatment. Marking a milestone, Ogsiveo becomes the inaugural drug sanctioned for addressing desmoid tumors, a rare subset of soft tissue sarcomas.
Desmoid tumors, though non-cancerous, exhibit local aggressiveness, potentially infiltrating nearby structures and organs. This invasion can lead to pain, restricted mobility, and a diminished quality of life. Historically, surgical removal has been the preferred treatment, but the high risk of tumor recurrence or other health complications post-removal has prompted increased exploration of systemic therapies in clinical trials.
The efficacy of Ogsiveo underwent evaluation in an international, multicenter, randomized, double-blind, placebo-controlled trial involving 142 adult patients with progressing desmoid tumors ineligible for surgery. Patients were randomly assigned to receive 150 milligrams (mg) of Ogsiveo or a placebo orally twice daily until disease progression or unacceptable toxicity. Progression-free survival, indicating the duration a person remains alive without cancer growth or spread, served as the primary efficacy outcome measure, with objective response rate (measuring tumor shrinkage) as an additional metric.
Results from the pivotal clinical trial revealed that Ogsiveo significantly improved progression-free survival compared to the placebo, both clinically and statistically. Moreover, the objective response rate differed markedly between the two groups, with a 41% response rate in the Ogsiveo group and 8% in the placebo group. Patient-reported pain assessment also favored the Ogsiveo group, supporting the progression-free survival findings.
Common side effects, observed in at least 15% of trial participants, included diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Ogsiveo secured Priority Review, signifying the FDA's commitment to expedite action within six months on applications where the drug, if approved, would significantly enhance safety or effectiveness compared to existing therapies. Additionally, Ogsiveo received FDA Fast Track and Breakthrough Therapy designations, along with Orphan-Drug designation for desmoid tumor (aggressive fibromatosis) treatment. The Orphan-Drug designation provides incentives to support and promote drug development for rare diseases. SpringWorks Therapeutics Inc. received FDA approval for Ogsiveo.