FDA Approves SpringWorks Therapeutics Treatment for Adult and Pediatric Patients with NF1-PN

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FDA has approved the SpringWorks Therapeutics treatment Gomekli (mirdametinib) for treatment of adult and pediatric patients aged two and older who have neurofibromatosis type 1 (NF1) with symptomatic plexiform neurofibromas (PN) that are not amenable to complete resection (1). SpringWorks, a commercial-stage biopharmaceutical company headquartered in Stamford, Conn., announced the approval on Feb. 11, 2025, adding that a marketing authorization application for mirdametinib has been validated by the European Medicines Agency and is currently under review, with a decision from the European Commission expected later in 2025.

Saqib Islam, SpringWorks CEO, said FDA’s approval was a win for NF1-PN patients in the United States.

“The NF1-PN patient community has a great need for more treatment options,” Islam said in the press release. “With today’s approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief. We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making Gomekli available in the US. I am proud that we are delivering on our commitment to patients with devastating diseases with our company’s second FDA approval in less than 18 months.”

SpringWorks said that Gomekli was approved under priority review by FDA—having previously been granted orphan drug and fast track designations—and that the company received a rare pediatric disease priority review voucher (1).

The future of the rare pediatric disease priority review voucher has been in some doubt; one such designation granted late in 2024 was to eladocagene exuparvovec-tneq (Kebilidi, PTC Therapeutics), for treating adult and pediatric patients with aromatic L-amino acid decarboxylase deficiency (2,3).

FDA’s approval of mirdametinib for patients with NF1-PN was based on results of a Phase IIb trial (ReNeu) that enrolled 114 patients nearly evenly split between adults and children (58 adult patients, 56 pediatric) (1). There are approximately 40,000 people living with NF1-PN in the US, according to SpringWorks, and most of them have not had previous access to an approved medication.

“Patients with NF1-PN often face significant challenges with their health and have had limited treatment options to manage this devastating condition,” Christopher Moertel, MD, medical director, pediatric neuro-oncology and neurofibromatosis programs and Kenneth and Betty Jayne Dahlberg Professor of Pediatrics, University of Minnesota, and lead investigator of the ReNeu trial, said in the SpringWorks press release. “It was very encouraging in the ReNeu trial to see that GOMEKLI provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment.”

“We are excited to celebrate the extraordinary milestone of our partners and long-term friends at SpringWorks for the NF1-PN community. This FDA approval shows the power of collaboration to advance innovative science for drugs that may otherwise not have been taken forward,” said Annette Bakker, PhD, Children’s Tumor Foundation CEO, in the release.

Gomekli, currently being manufactured in both 1 mg and 2 mg capsules, as well as a 1 mg tablet for oral suspension that dissolves easily in water, is expected to be available in the US through a specialty pharmacy and distributor network by the end of February 2025.

References

1. SpringWorks Therapeutics. SpringWorks Therapeutics Announces FDA Approval of Gomekli (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN. Press Release. Feb. 11, 2025.
2. National Organization for Rare Disorders. Congress: Reauthorize the Rare Pediatric Disease Priority Review Voucher Program. Rarediseases.org, July 23, 2024.
3. FDA. FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency. Press Release. Nov. 14, 2024.