Brussels, May 2025 — SpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on rare diseases and oncology, announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending conditional marketing authorization for mirdametinib, a MEK inhibitor, to treat symptomatic, inoperable plexiform neurofibromas (PN) in pediatric and adult patients with neurofibromatosis type 1 (NF1) aged two and older.
The European Commission is expected to make a final decision on the approval by Q3 2025.
“This positive CHMP opinion brings us one step closer to delivering mirdametinib to patients in Europe. We are committed to making this important therapy available to both children and adults living with NF1-PN as soon as possible.”
— Saqib Islam, CEO of SpringWorks.
Neurofibromatosis type 1 (NF1) is a rare genetic disorder that affects approximately 135,000 people in the EU (3 in every 10,000). Up to 50% of these patients develop plexiform neurofibromas, tumors that grow along peripheral nerves, often causing pain, disfigurement, and functional impairment.
These tumors can also evolve into malignant peripheral nerve sheath tumors, a highly aggressive and life-threatening condition. Due to their infiltrative nature, approximately 85% of PN cases are considered inoperable, making new therapeutic options critically needed.
“NF1-PN is a life-altering condition with few treatment options. Mirdametinib, if approved, would offer an essential non-surgical option for patients.”
—Dr. Ignacio Blanco, Chairman at the National Reference Center for Adult Patients with Neurofibromatosis in Spain.
The CHMP opinion is based on compelling data from the Phase 2b ReNeu trial, which enrolled 114 patients (58 adults, 56 children) with inoperable symptomatic NF1-PN.
Key findings include:
The treatment, delivered orally as capsules or dispersible tablets, followed a 3-weeks-on, 1-week-off dosing schedule at a maximum dose of 4 mg twice daily.
Mirdametinib demonstrated a manageable safety profile in both adults and children.
These findings, presented at the 2024 ASCO Annual Meeting, support mirdametinib’s potential as a first-in-class, disease-modifying treatment for NF1-PN in Europe.
Mirdametinib is a selective MEK1/2 inhibitor designed to block the MAPK pathway, which is dysregulated in NF1 due to mutations in the NF1 gene, a tumor suppressor encoding neurofibromin.
Currently, mirdametinib is approved in the U.S. for adults and children (2+ years) with symptomatic, inoperable NF1-PN. It has received Orphan Drug Designation from both the FDA and European Commission for this indication.
SpringWorks Therapeutics is rapidly emerging as a leader in rare disease and oncology drug development. The company is already marketing the first FDA-approved therapy for desmoid tumors and has brought mirdametinib to U.S. patients with NF1-PN.
With EMA approval, SpringWorks would expand its geographic reach in Europe, offering a lifeline to thousands of NF1 patients who have long had limited or no medical options.
Conclusion
The CHMP’s recommendation marks a critical milestone for both SpringWorks and the NF1 patient community in Europe. Should the European Commission follow through with final approval later this year, mirdametinib may soon become the first widely available treatment for NF1-associated plexiform neurofibromas across the EU, representing a breakthrough in precision oncology for rare genetic conditions.