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【FDA Approval Alert】SpringWorks’s mirdametinib (Gomekli) Approved for NF1-Associated Plexiform Neurofibromas (February 11, 2025)
On February 11, 2025, the U.S. FDA approved mirdametinib (Gomekli) for patients aged 2 years and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). Approval was based on the ReNeu trial (NCT03962543).
Disease Overview: NF1-Associated Plexiform Neurofibromas
NF1 is a hereditary tumor predisposition syndrome. PN are benign nerve sheath tumors that may cause pain, functional impairment, and disfigurement. Targeted therapies offer non-surgical options for inoperable tumors.
Treatment Summary
- Drug Name: mirdametinib (Gomekli)
- Company: SpringWorks Therapeutics, Inc.
- Approval Date: February 11, 2025
- Indication: Symptomatic, inoperable NF1-associated PN (≥2 years old)
- Mechanism of Action: MEK inhibitor
- Pivotal Trial: ReNeu (NCT03962543)
- Key Efficacy:
- Adults: ORR 41% (95% CI: 29–55)
- Pediatrics: ORR 52% (95% CI: 38–65)
Patient-Friendly Summary
Gomekli blocks tumor growth signals, shrinking inoperable plexiform neurofibromas and providing a new oral treatment option.
M&A Information
- Subsidiary: Not applicable (SpringWorks in-house development)
- Acquisition Date: Not applicable
- Development Stage at Acquisition: Not applicable
- Investor Information: Public Investors (NASDAQ: SWTX)
Source
This approval information is based on data from Drugs@FDA (U.S. FDA) and AACR releases.
