The approval comes days after Germany’s Merck KGaA confirmed it was in advanced talks to acquire SpringWorks.
The FDA on Tuesday signed off on SpringWorks Therapeutics’ mirdametinib—now to carry the brand name Gomekli—for the treatment of patients with a specific type of neurofibromatosis.
The drug, which is taken orally, can be used in both adults and children aged 2 years and above—the first medicine indicated for both adult and pediatric populations with neurofibromatosis type 1 with symptomatic plexiform neurofibromas (NF1-PN), according to the company’s statement.
Gomekli’s approval was backed by data from the Phase IIb ReNeu study, which enrolled 58 adults and 56 children with NF1-PN. The primary outcome, objective response rate as assessed by a blinded independent review board, was 41% in the adult cohort and 52% in pediatric patients. These response results were accompanied by “deep and durable” reductions in tumor volume, according to SpringWorks, as well as “early and sustained” improvements in pain and quality of life.
“The NF1-PN patient community has a great need for more treatment options,” SpringWorks CEO Saqib Islam said in a statement, noting that Gomekli’s approval provides these patients with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief.” Gomekli will be available through specialty distributors and pharmacies “within two weeks,” as per Tuesday’s announcement.
Neurofibromatosis is a highly diverse condition that refers to the development of mostly benign tumors affecting the brain, spinal cord and nerves. NF1-PN, specifically, afflicts around 40,000 patients in the U.S. The mostly benign tumors that develop in patients with NF1-PN carry an extra risk of transforming into malignant peripheral nerve sheath tumors.
With the approval of Gomekli, SpringWorks will now go head-to-head with AstraZeneca’s drug Koselugo, which was approved in 2020 for NF1-PN. Koselugo is a high-growth asset for the pharma, surging 96% year-on-year to earn $631 million in 2024. SpringWorks is challenging AstraZeneca’s first-to-market advantage with a broader label. Unlike Gomekli, Koselugo is indicated only for pediatric NF1-PN patients.
Gomekli’s regulatory win also comes just days after German pharma Merck KGaA confirmed that it is in talks with SpringWorks regarding a potential acquisition. The parties have yet to enter into any legally binding agreements and the “there is no certainty that any transaction will materialize,” Merck KGaA insisted.
Still, news of the acquisition sent shares of SpringWorks soaring 34% in its aftermath, bringing the biotech’s market value to approximately $4 billion at market close on Monday. Gomekli is only SpringWorks’ second FDA-approved drug, after Ogsiveo, a treatment for desmoid tumors approved in November 2023.
