STAMFORD, Conn. - SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a biopharmaceutical company, has announced that the European Medicines Agency (EMA) has accepted its Marketing Authorization Application (MAA) for nirogacestat, a proposed treatment for adults with desmoid tumors. This milestone marks a significant step towards the potential availability of the first authorized therapy for this condition in the European Union (EU).
The MAA is supported by data from the Phase 3 DeFi trial, where nirogacestat showed a 71% reduction in the risk of disease progression compared to placebo. The trial's results, published in the New England Journal of Medicine, highlighted a median progression-free survival (PFS) that was not reached in the nirogacestat arm against 15.1 months in the placebo arm. Additionally, the objective response rate was 41% with nirogacestat, significantly higher than the 8% with placebo.
Desmoid tumors are rare and can be life-threatening when they affect vital structures. They are most commonly diagnosed in patients between the ages of 20 and 44, with a higher prevalence in females. Current estimates suggest an incidence of 3-5 cases per million per year in the EU. Despite not being prone to metastasize, desmoid tumors often recur after surgical removal, leading experts to recommend systemic therapies as a first-line intervention.
Nirogacestat, which has already been granted Orphan Drug designation by the European Commission for the treatment of soft tissue sarcoma, has a manageable safety profile. The most common adverse events in the trial included diarrhea, ovarian toxicity, and rash. In November 2023, the U.S. Food and Drug Administration approved nirogacestat for the treatment of adults with progressing desmoid tumors who require systemic therapy.
SpringWorks Therapeutics CEO Saqib Islam expressed the company's commitment to working with the EMA to bring this therapy to patients in the EU. Dr. Bernd Kasper from the University of Heidelberg, Germany, and the Principal Investigator of the DeFi trial, also emphasized the potential of nirogacestat to change clinical practice for treating desmoid tumors.
This announcement is based on a press release statement from SpringWorks Therapeutics, Inc. The company continues to explore nirogacestat's potential in other areas, such as ovarian granulosa cell tumors and multiple myeloma, in collaboration with industry and academic leaders.
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