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Koselugo (selumetinib)
Selumetinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).
Selumetinib (Koselugo), marketed by AstraZeneca LP, is a MEK1/2 inhibitor approved for treating Neurofibromatosis Type 1 with Symptomatic, Inoperable Plexiform Neurofibromas. Its key strength lies in being the first and currently only FDA-approved therapy for this indication, providing a significant competitive advantage. The primary risk is the key composition patent expiry in 2028, which could lead to increased competition from generic versions and other MEK inhibitors like trametinib, cobimetinib, and adagrasib.
At a glance
| Generic name | selumetinib |
|---|---|
| Sponsor | ASTRAZENECA LP |
| Drug class | Kinase Inhibitor [EPC] |
| Target | AP2-associated protein kinase 1 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2020 |
Mechanism of action
Selumetinib works by inhibiting the MEK1/2 proteins, which are upstream regulators of the extracellular signal-related kinase (ERK) pathway. This pathway is often activated in different types of cancers. By inhibiting MEK1/2, selumetinib reduces the activation of the ERK pathway, leading to a decrease in neurofibroma numbers, volume, and proliferation.
Approved indications
- Neurofibromatosis Type 1 with Symptomatic, Inoperable Plexiform Neurofibromas
Common side effects
- Vomiting
- Abdominal pain
- Diarrhea
- Nausea
- Stomatitis
- Rash (all)
- Dry skin
- Paronychia
- Pruritus
- Dermatitis
- Musculoskeletal pain
- Fatigue
Drug interactions
- Strong or Moderate CYP3A4 Inhibitors or Fluconazole
- Strong or Moderate CYP3A4 Inducers
- Vitamin E (supplemental)
Key clinical trials
- AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors (PHASE1, PHASE2)
- A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma (PHASE3)
- Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma (PHASE1, PHASE2)
- Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial (PHASE2)
- Cediranib Maleate and Selumetinib Sulfate in Treating Patients With Solid Malignancies (PHASE1)
- A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in Patients With Recurrent or Progressive Low-Grade Glioma (PHASE3)
- Testing the Use of the Combination of Selumetinib and Olaparib or Selumetinib Alone Targeted Treatment for RAS Pathway Mutant Recurrent or Persistent Ovarian and Endometrial Cancers, A ComboMATCH Treatment Trial (PHASE2)
- A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Koselugo CI brief — competitive landscape report
- Koselugo updates RSS · CI watch RSS
- ASTRAZENECA LP portfolio CI