European Commission Approves SpringWorks Therapeutics' OGSIVEO for Desmoid Tumors Treatment

SpringWorks Therapeutics has received European Commission approval for Ogsiveo (nirogacestat) in desmoid tumors. The FDA-approved therapy is for adult patients with progressing desmoid tumors requiring systemic treatment. The company has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with ongoing developments including Nirogacestat for ovarian granulosa cell tumors and Mirdametinib Phase 2b ReNeu trial in NF1-PN.

SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, has received a significant milestone with the European Commission's (EC) approval of Ogsiveo (nirogacestat) for the treatment of adults with progressing desmoid tumors. This approval marks the first and only therapy to be granted marketing authorization in the European Union (EU) for this condition.

Desmoid tumors are rare, locally aggressive tumors that form in the connective tissues of the body, causing severe pain, limited function, and loss of mobility. The EC approval is based on the results of the Phase 3 DeFi trial, which enrolled 142 adult patients with progressing desmoid tumors. The trial demonstrated a statistically significant improvement in progression-free survival (PFS) with a 71% reduction in the risk of disease progression compared to placebo. The confirmed objective response rate (ORR) was 41% with Ogsiveo versus 8% with placebo, highlighting the therapy's efficacy.

The approval of Ogsiveo is a significant advancement for patients with desmoid tumors, their families, and physicians in Europe. Desmoid tumor experts now recommend medical therapy as first-line intervention instead of surgery for most tumor locations requiring treatment. This approval follows the EC's recent approval of Ogsiveo for patients with NF1-PN, underscoring SpringWorks' commitment to the rare tumor patient community.

SpringWorks Therapeutics is dedicated to improving the lives of patients with rare tumors. The company's diversified targeted therapy pipeline includes ongoing developments such as Nirogacestat for ovarian granulosa cell tumors and the Mirdametinib Phase 2b ReNeu trial in NF1-PN. The successful approval of Ogsiveo in the EU further positions SpringWorks as a leader in the rare tumor therapeutic landscape.

References:
[1] https://www.globenewswire.com/news-release/2025/08/18/3135068/0/en/European-Commission-Grants-Approval-of-OGSIVEO-nirogacestat-for-the-Treatment-of-Adults-with-Desmoid-Tumors.html