While tumor development in neurofibromatosis types 1 and 2 (NF1 and NF2) is typically benign, physicians are particularly concerned about malignant transformation (notably in NF1) and long-term quality of life in patients. These concerns are especially significant when tumors involve the central nervous system, including gliomas or meningiomas, where even benign growth can cause substantial neurological morbidity.

Today, we are taking a look at what's recently changed regarding neurofibromatosis in relation to clinical guidance, FDA approvals, and clinical trials.

Current Neurofibromatosis Guidelines

There are currently no clinical guidelines covering both types of neurofibromatosis. There is a guideline from the European Reference Network (ERN) focusing on tumor management for neurofibromatosis type 1 (NF1), but it remains to be seen if there will be a similar guideline issued for neurofibromatosis type 2 (NF2), or additional guidelines from U.S.-based medical societies.

We were, however, able to identify a clinical practice resource issued by the American College of Medical Genetics and Genomics (ACMG) on the care of adults with neurofibromatosis type 1, which you can find linked below.

Tumor Management in Neurofibromatosis Type 1

  • European Reference Network (ERN)
  • Published: January 2023

Care of Adults with Neurofibromatosis Type 1

  • American College of Medical Genetics and Genomics (ACMG)
  • Published: April 2018
Recent FDA Approvals for Neurofibromatosis

Below are the recent medications and therapies recently approved by the FDA that we anticipated being incorporated into future clinical guidelines: 

Koselugo (selumetinib)

  • AstraZeneca
  • Approved: November 2025
  • For the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

Koselugo (selumetinib)

  • AstraZeneca
  • Approved: September 2025
  • For pediatric patients 1 year of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

Gomekli (mirdametinib)

  • SpringWorks Therapeutics
  • Approved: February 2025
  • For the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
Ongoing Clinical Trials Related to Neurofibromatosis

The following are a pair of ongoing, Phase 3 clinical trials based in the United States that we anticipate may impact any future neurofibromatosis clinical guidelines.

Sign up for alerts to keep up with the latest guidelines and insights.

Copyright © 2026 Guideline Central, all rights reserved.