SpringWorks Therapeutics announced today that it anticipates receiving an opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on its marketing authorization application for nirogacestat in the second quarter of 2025. The application seeks approval for the oral gamma secretase inhibitor in the treatment of adults with desmoid tumors.
The Connecticut-based biopharmaceutical company is seeking to expand the global footprint of nirogacestat, which is already approved and commercialized in the United States under the brand name OGSIVEO. The therapy represents a significant advancement as the first and only FDA-approved medicine specifically for adults with desmoid tumors.
Understanding Desmoid Tumors and Treatment Needs
Desmoid tumors, also known as aggressive fibromatosis, are rare soft tissue tumors that can occur in virtually any part of the body. While not metastatic, these tumors can be locally aggressive, causing pain, functional limitations, and significant morbidity through their invasion of surrounding structures. Prior to dedicated therapies, treatment options were limited to surgery, radiation, and off-label use of various medications with inconsistent results.
The development of nirogacestat addresses a significant unmet need for patients with this rare condition. Desmoid tumors affect approximately 1,000-1,500 new patients annually in the United States, with similar prevalence rates expected in European populations.
Expanding Global Access to Innovative Therapies
"The anticipated CHMP opinion represents an important milestone in our efforts to bring nirogacestat to patients with desmoid tumors throughout Europe," said a SpringWorks representative in the announcement. The company did not provide specific details on the clinical data submitted to the EMA, though the application likely includes results from the phase 3 DeFi trial that supported FDA approval.
The DeFi trial demonstrated that nirogacestat significantly improved progression-free survival compared to placebo and showed meaningful reductions in pain and other symptoms that impact patients' daily functioning.
SpringWorks' Growing Rare Disease Portfolio
SpringWorks has established itself as a leader in developing therapies for rare diseases and cancer. Beyond nirogacestat, the company has successfully developed GOMEKLI (mirdametinib), the first and only FDA-approved medicine for both adults and children with neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN).
The company continues to advance a diverse portfolio of novel targeted therapy product candidates for patients with both solid tumors and hematological cancers, reinforcing its commitment to addressing high unmet medical needs in oncology and rare diseases.
Regulatory Pathway and Timeline
The CHMP opinion, expected in Q2 2025, will serve as a recommendation to the European Commission, which makes the final decision on marketing authorization in the European Union. If approved, nirogacestat would become available to adult patients with desmoid tumors across EU member states.
The regulatory review process in Europe typically takes 12-14 months from submission to final decision, suggesting that SpringWorks likely submitted its application in early to mid-2024.
Market Implications
Approval in the European market would significantly expand the potential patient population for nirogacestat. For SpringWorks, which has transformed from a clinical-stage to a commercial-stage company in recent years, European approval would represent another important commercial milestone.
The company's stock (Nasdaq: SWTX) has shown resilience as investors monitor the progress of both its commercial products and development pipeline. European approval would potentially strengthen the company's revenue outlook and global presence in the rare disease space.
Mechanism of Action
Nirogacestat works by inhibiting gamma secretase, an enzyme complex that plays a role in the Notch signaling pathway, which is implicated in the development and progression of desmoid tumors. By targeting this specific molecular mechanism, the therapy addresses the underlying biology of the disease rather than simply managing symptoms.
This targeted approach exemplifies the shift toward precision medicine in rare disease treatment, where therapies are designed to address specific molecular drivers of disease pathology.
As SpringWorks awaits the CHMP opinion, patients with desmoid tumors in Europe may soon have access to the same innovative therapy that has already begun to transform the treatment landscape for this rare condition in the United States.
