FDA Approves SpringWorks’ Gomekli for Adult and Pediatric NF1-PN Patients

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 resection13.

Gomekli is the first and only medicine approved for both adults and children with NF1-PN14.

The approval is based on positive data from the Phase 2b ReNeu trial, which showed robust objective response rates, deep and durable reductions in tumor volume, and a manageable safety profile14.

In the ReNeu trial, Gomekli demonstrated a 41% objective response rate in adults and 52% in children4.

Patients experienced early and sustained significant improvements in pain and quality of life4.

SpringWorks was granted a rare pediatric disease priority review voucher by the FDA with this approval14.

Gomekli will be available through specialty distributors and pharmacies within two weeks of the approval7.

This approval marks SpringWorks' second FDA-approved drug in less than 18 months, following Ogsiveo for desmoid tumors in November 20237.

Gomekli will compete with AstraZeneca's Koselugo, which was approved in 2020 for pediatric NF1-PN patients7.

The approval comes shortly after German pharma Merck KGaA confirmed it was in talks with SpringWorks regarding a potential acquisition7.

Sources:

1. 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

3. https://www.pharmacytimes.com/view/fda-approves-mirdametinib-for-treatment-of-adults-children-with-nf1-pn

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

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