FDA Approves SpringWorks Therapeutics’ Gomekli for Neurofibromatosis Type 1, Symptomatic Plexiform Neurofibromas
Approval makes Gomekli the first treatment to be approved for both adults and pediatric patients for neurofibromatosis type 1, a genetic disorder that causes noncancerous tumors to grow on nerves throughout the body.
Image Credit: Adobe Stock Images/solvod
The FDA has approved SpringWorks Therapeutics’ Gomekli (mirdametinib), an MEK inhibitor, for the treatment of adult and pediatric patients over two years of age with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas (PN) not amenable to complete resection. According to the company, the approval marks the first treatment to be available for both adults and children with NF1, a genetic disorder that causes noncancerous tumors to grow on nerves throughout the body. Approval was based on results from the Phase IIb ReNeu trial.1
“The NF1-PN patient community has a great need for more treatment options. 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,” said Saqib Islam, CEO, SpringWorks, in a press release. “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.”
The open-label, multicenter, pivotal ReNeu trial trial enrolled a total of 114 individuals, including 58 adults and 56 pediatric patients. The primary endpoint of the study was confirmed objective response rate (ORR), as assessed by blinded independent central review. Secondary efficiency endpoints included duration of response and change from baseline to prespecified cycle 13 for patient-reported outcome or parent proxy-reported outcome measures of worst tumor pain severity.1,2
Gomekli demonstrated an ORR of 41% in adults and 52% in children, with durable tumor reductions and improvements in pain and quality of life. Additionally, 88% of adults and 90% of children with a confirmed response reported a response that lasted at least one full year. Further, 50% of adults and 48% of children had a response of at least two years.
Common adverse events (AEs) in adults were rash, diarrhea, nausea, musculoskeletal pain, vomiting and fatigue. Common AEs in children included rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. The treatment demonstrated a manageable safety and tolerability profile.1
“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, CEO, the Children’s Tumor Foundation, in the press release. "When industry, researchers, and organizations like ours driving treatment innovation join forces, scientific progress moves faster, and patients gain access to the therapies they need. Every treatment approval is hard-won, built on research, persistence, and partnership. Today, that work delivers a critical new option for NF patients of all ages.”
According to SpringWorks, an estimated 100,000 children and adults in the United States are currently living with NF1. Patients with NF1 have anywhere from a 30% to 50% chance of developing NF1PN. It is estimated that there are around 40,000 people in the United States living with NF1-PN. Of these patients, a majority have not previously had an approved treatment available.1
“NF1-PN is a complex, devastating disease that affects not only individual patients, but entire families. Treatment advances are crucial to achieving better outcomes for patients and this FDA approval offers hope for NF patients and their families,” said Kim Bischoff, executive director, NF Network, in the press release.
Reference
1. SpringWorks Therapeutics Announces FDA Approval of GOMEKLI™ (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN. SpringWorks. February 11, 2025. Accessed February 12, 2025. https://ir.springworkstx.com/news-releases/news-release-details/springworks-therapeutics-announces-fda-approval-gomeklitm
2. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. ASCOPubs. Accessed February 12, 2025. https://ascopubs.org/doi/10.1200/JCO.24.01034
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