SpringWorks’ drug nears EU approval for neurofibromatosis treatment

Published 23/05/2025, 13:06
SpringWorks’ drug nears EU approval for neurofibromatosis treatment

STAMFORD, Conn. - SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), currently trading at $46.27 with a market capitalization of $3.49 billion, has announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the conditional marketing authorization of mirdametinib for treating plexiform neurofibromas in patients with neurofibromatosis type 1 (NF1). According to InvestingPro analysis, the company’s stock is trading near its Fair Value, with impressive revenue growth of over 730% in the last twelve months. The European Commission is expected to make a final decision on this recommendation in the third quarter of 2025.

Mirdametinib, if approved, will be the first therapy in the European Union authorized for both children and adults with NF1-related plexiform neurofibromas (NF1-PN). The drug will be available in 1 and 2 mg capsules, as well as a 1 mg dispersible tablet.

Neurofibromatosis type 1 is a genetic disorder affecting around 135,000 people in the EU. Patients with NF1 have a significant risk of developing plexiform neurofibromas, which are tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain, and functional impairment. These tumors are often inoperable and can lead to malignant peripheral nerve sheath tumors. SpringWorks maintains strong financial health with a current ratio of 4.33, indicating robust liquidity to support its commercialization efforts. For detailed analysis of SpringWorks’ market position and growth potential, investors can access comprehensive research through InvestingPro, which offers exclusive insights and financial metrics.

The CHMP’s positive opinion is based on results from the Phase 2b ReNeu trial, which showed a confirmed objective response rate of 41% in adults and 52% in children. Patients also reported significant improvements in pain and quality of life. The safety profile of mirdametinib was considered manageable, with the most common adverse events being rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. The company’s strong gross profit margin of 93.23% suggests efficient cost management in its drug development programs.

Mirdametinib has already been approved in the United States for the treatment of symptomatic, inoperable plexiform neurofibromas in NF1 patients aged 2 years and older. The drug’s development reflects SpringWorks Therapeutics’ commitment to addressing severe rare diseases and cancers.

The information in this article is based on a press release statement from SpringWorks Therapeutics.

In other recent news, SpringWorks Therapeutics has been in the spotlight with several key developments. The company recently held its annual stockholders’ meeting, where Alan Fuhrman, Julie Hambleton, M.D., and Daniel S. Lynch, M.B.A. were elected as Class III directors. Additionally, stockholders ratified Ernst & Young LLP as the independent registered public accounting firm for the fiscal year ending December 31, 2025. In analyst updates, TD Cowen downgraded SpringWorks from Buy to Hold and adjusted the price target to $47.00, citing the proposed acquisition by Merck KGaA as a factor. Meanwhile, H.C. Wainwright maintained a Buy rating with a $74.00 price target, focusing on the anticipated European Medicines Agency decision on SpringWorks’ drug nirogacestat in 2025. Barclays also reiterated an Overweight rating with a $63.00 price target, emphasizing the potential EU approval of Ogsiveo, which could influence the acquisition dynamics with Merck KGaA. The acquisition is expected to bolster Merck’s oncology portfolio, particularly in rare tumors, and expand their market presence. These developments highlight strategic moves for SpringWorks as it advances its pipeline and considers its future with Merck KGaA.

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