Springworks neurofibromatosis drug OKd

 – GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN –

– Approval based on positive data from Phase 2b ReNeu trial, which showed GOMEKLI treatment resulted in robust ORR, deep and durable reductions in tumor volume, and a manageable safety profile –

– SpringWorks granted rare pediatric disease priority review voucher by the FDA –

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