Koselugo - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation

selumetinib

Opinion

On 18 September 2025 the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Koselugo. The marketing authorisation holder for this medicinal product is AstraZeneca AB.

The CHMP adopted a change to the existing indication as follows¹:

Koselugo as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult and paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and older above.

For information, the full indication for Koselugo will be as follows:

Koselugo as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult and paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and older.

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

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¹New text in bold, removed text as strikethrough

CHMP post-authorisation summary of positive opinion for Koselugo (EMAVR0000245231)

Adopted Reference Number: EMADOC-1700519818-2413725

Key facts

• Name of medicine:
  Koselugo
• EMA product number:
  EMEA/H/C/005244
• Active substance:
  selumetinib sulfate
• International non-proprietary name (INN) or common name:
  selumetinib
• Therapeutic area (MeSH):
  Neurofibromatosis 1
• Additional monitoring:

  This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

• Conditional approval:

  This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.

• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation holder:
  AstraZeneca AB
• Date of opinion:
  18/09/2025
• Status:
  Positive