Selumetinib
Orphan DrugFDA Approved, EMA Approved
Contents
Description
Selumetinib is a kinase inhibitor that targets MEK1 and MEK2. It was approved for pediatric patients with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas. The drug represents the first approved treatment specifically for this rare genetic condition.
Indications & Therapeutic Use
neurofibromatosis type 1, plexiform neurofibromas
Linked Diseases:
intellectual disability
F79D008607
Global Availability (4 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Selumetinib
| Generic Name | selumetinib |
| Brands | 1 brand available |
| Active Ingredient | selumetinib sulfate |
| Drug Class | neurofibromatosis type 1 |
| Manufacturer | AstraZeneca |
| Dosage Forms | Oral capsules, 10mg and 25mg |
| Medical Code | L01EE04 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT01362803 |
| Countries | 4 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations5 Validated Nodes