Mirdametinib FDA Approval Status
Last updated by Judith Stewart, BPharm on March 19, 2024.
FDA Approved: No
Generic name: mirdametinib
Company: SpringWorks Therapeutics, Inc.
Treatment for: Neurofibromatosis
Mirdametinib is an investigational MEK inhibitor in development for the treatment of patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).
- Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene. The NF1 gene encodes for neurofibromin, a key suppressor of the MAPK pathway which is a key signaling network that regulates cell growth and survival, and plays a central role in multiple oncology and rare disease indications when genetically altered. NF1 patients are at risk of developing plexiform neurofibromas, which are tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain and functional impairment.
- Mirdametinib works by inhibiting MEK1 and MEK2, which occupy pivotal positions in the MAPK pathway.
- The U.S. Food and Drug Administration (FDA) has granted mirdametinib Orphan Drug designation and Rare Pediatric Disease designation for the treatment of NF1, and granted Fast Track designation for the treatment of patients ≥ 2 years of age with NF1-PN that are progressing or causing significant morbidity.
Development timeline for mirdametinib
Further information
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