USANew treatments and approvals
Mirdametinib approved in the USA for NF1-related plexiform neurofibromas
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The US drug agency (FDA) has approved oral mirdametinib for patients with NF1. The drug is for patients aged 2 years and older. The patient must have a plexiform neurofibroma that causes symptoms. This tumor must be one that cannot be completely removed by surgery. The approval is based on the ReNeu study. In this study, tumor shrinkage was seen in about 41% of adults and in 52% of children. Common side effects include rash and diarrhea.
What does it mean for patients and families?
A new drug option has emerged for both children and adults with NF1. Availability in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) licensing and SGK (Social Security Institution) reimbursement processes.
Source
The ASCO Post: FDA Approves Mirdametinib for Neurofibromatosis Type 1
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Neurofibromatosis type 1 (NF1)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.