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STAMFORD, Conn. - SpringWorks Therapeutics announced Friday that the European Commission has granted conditional marketing authorization for EZMEKLY (mirdametinib) to treat symptomatic, inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1) aged 2 years and above. The news comes as SpringWorks stock has gained over 30% year-to-date, according to InvestingPro data, with investors anticipating the company’s next earnings report on July 30.
EZMEKLY becomes the first therapy approved in the European Union for both adults and children with NF1-PN, a rare genetic disorder affecting approximately 135,000 people across the EU. This milestone approval has contributed to SpringWorks’ impressive 730% revenue growth over the last twelve months, as reported by InvestingPro, which offers comprehensive financial analysis and additional ProTips for this emerging biotech player.
The approval is based on results from the Phase 2b ReNeu trial, which demonstrated an objective response rate of 41% in adults and 52% in children. Among responding patients, 88% of adults and 90% of children maintained their response for at least 12 months.
"This approval represents an important advance, especially for adults who previously did not have an approved treatment," said Ignacio Blanco, Chairman of the National Reference Center for Adult Patients with Neurofibromatosis at Hospital Universitari Germans Trias i Pujol, Spain.
NF1-PN tumors grow along the peripheral nerve sheath and can cause disfigurement, pain and functional impairment. Approximately 85% of these tumors are considered not amenable to complete surgical resection.
The most common adverse reactions in adults included dermatitis acneiform (83%), diarrhea (55%), and nausea (55%), while children most frequently experienced increased blood creatine phosphokinase (59%), diarrhea (53%), and dermatitis acneiform (43%).
EZMEKLY is available in capsule form and as a dispersible tablet that dissolves in water for patients unable to swallow pills.
The treatment received FDA approval in the United States, where it is marketed under the brand name GOMEKLI. SpringWorks maintains a strong financial position with a healthy current ratio of 4.33 and minimal debt, according to InvestingPro analysis, positioning the company well for commercial expansion in both markets. Discover more detailed insights and metrics in the exclusive InvestingPro Research Report, available for over 1,400 US stocks.
In other recent news, Merck KGaA has completed its acquisition of SpringWorks Therapeutics for $47 per share, making SpringWorks a wholly owned subsidiary. This merger follows regulatory clearances, including approvals from Germany’s antitrust authority and the expiration of the Hart-Scott-Rodino waiting period in the U.S. Additionally, SpringWorks announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended nirogacestat for treating desmoid tumors, which could become the first authorized therapy for this condition in the European Union. The company also received a recommendation for mirdametinib’s conditional marketing authorization for treating neurofibromatosis type 1-related plexiform neurofibromas, with a decision expected in the third quarter of 2025. SpringWorks held its annual stockholders’ meeting, where key resolutions, including the election of directors and the ratification of Ernst & Young LLP as the independent accounting firm, were passed. These developments highlight significant progress for SpringWorks in both its merger activities and drug approvals.
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