Mirdametinib

Orphan DrugFDA Approved (2025)

Description

An oral MEK1/2 inhibitor for adults and children aged 2 years and older with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. FDA-approved February 2025.

Indications & Therapeutic Use

Neurofibromatosis type 1 (plexiform neurofibromas)

Linked Diseases:

Neurofibromatosis Type 1
LD2D.10D009456

Evidence: FDA-approved indication

Global Availability (1 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus
Standard Marketing AuthorizationApproved

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Mirdametinib
Generic Namemirdametinib
Brands1 brand available
Active Ingredientmirdametinib
Drug ClassNeurofibromatosis type 1 (plexiform neurofibromas)
ManufacturerSpringWorks Therapeutics
Orphan StatusYes — Orphan Drug
Cold ChainNot Required
Reg. StatusFDA Approved (2025)
Countries1 countries
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes