EMA Recommends Approval of Nirogacestat for Desmoid Tumors

The European Medicines Agency's (EMA) Committee for Human Medicinal Products has issued a favorable recommendation for the approval of Nirogacestat, aimed at treating adults with progressive desmoid tumors who require systemic therapy. Desmoid tumors, which arise from the proliferation of differentiated myofibroblasts, are classified as benign soft tissue tumors and are known for their rarity and heterogeneity, complicating treatment options.

Nirogacestat was previously granted approval in the United States in November 2023. The applicant, Spring Works Therapeutics Ireland Limited, plans to introduce the Nirogacestat-containing medication Ogsiveo® in European markets, available in film-coated tablets with dosages of 50 mg, 100 mg, and 150 mg.

This medication operates as a ?-secretase inhibitor, targeting a novel mechanism. By inhibiting this enzyme, Nirogacestat prevents the proteolytic activation of Notch receptors, which play a significant role in tumor formation and growth.

According to the EMA, Ogsiveo demonstrated a statistically significant improvement in progression-free survival (PFS) compared to placebo in a Phase III clinical trial involving adults with progressive desmoid tumors. The most common adverse effects reported include diarrhea, rash, ovarian toxicity in women of childbearing age, nausea, fatigue, hypophosphatemia, headaches, and mucosal inflammation.

The final decision regarding Nirogacestat's approval in the European Union now rests with the European Commission. Typically, this process is a formality, as the Commission generally aligns with the EMA's recommendations.