SpringWorks Therapeutics announced on February 11, 2025, that the U.S. Food and Drug Administration (FDA) approved GOMEKLI (mirdametinib) for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN, addressing a significant unmet medical need for approximately 40,000 people in the United States. The approval was based on positive data from the Phase 2b ReNeu trial, which demonstrated a 41% objective response rate (ORR) in adults and 52% in children, with deep and durable tumor volume reductions.
With this approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA, a valuable asset. GOMEKLI is expected to be available through a specialty pharmacy and specialty distributor network in the United States within two weeks.
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