SpringWorks Therapeutics Secures FDA Approval for Gomekli to Treat NF1-Related Tumors

The FDA has approved Gomekli (mirdametinib) for the treatment of adult and pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection15.

Gomekli is the first and only medicine approved for both adults and children with NF1-PN8.

The approval was based on positive data from the Phase 2b ReNeu trial, which showed significant tumor shrinkage in both adult and pediatric patients18.

In the clinical trial, 41% of adults and 52% of children saw their tumors shrink by at least 20% within two to six months of treatment2.

Gomekli works by blocking enzymes that help tumors grow, which helps shrink them and slow their spread2.

With this approval, SpringWorks received a rare pediatric disease priority review voucher from the FDA58.

The approval comes amid talks of a potential acquisition of SpringWorks by German pharma company Merck KGaA1.

Gomekli will compete with AstraZeneca's Koselugo, which was approved in 2020 for NF1-PN but is only indicated for pediatric patients1.

NF1 is a nervous system disorder that affects about 100,000 people in the United States, with 30% to 40% of children with NF1 potentially developing plexiform neurofibromas2.

This is SpringWorks' second FDA-approved drug, following the approval of Ogsiveo for desmoid tumors in November 20231.

Sources:

1. https://www.biospace.com/fda/springworks-wins-fda-nod-for-neurofibromatosis-drug-tees-up-challenge-to-astrazeneca

2. https://www.webmd.com/pain-management/news/20250212/fda-approves-new-treatment-for-nf1-related-tumors

5. https://ir.springworkstx.com/news-releases/news-release-details/springworks-therapeutics-announces-fda-approval-gomeklitm

8. https://www.globenewswire.com/news-release/2025/02/11/3024643/0/en/SpringWorks-Therapeutics-Announces-FDA-Approval-of-GOMEKLI-mirdametinib-for-the-Treatment-of-Adult-and-Pediatric-Patients-with-NF1-PN.html