AstraZeneca PLC — Koselugo EMA EPAR public assessment report (EMEA/H/C/005244, 2021)
AstraZeneca PLC · 2021 · EMA EPAR public assessment report · company profile →
Report summary
This report summarizes the marketing authorization for Koselugo (selumetinib) for pediatric NF1 patients with symptomatic, inoperable plexiform neurofibromas. Selumetinib, a MEK1/2 inhibitor, demonstrated significant efficacy in the SPRINT Phase II study, achieving a 66% objective response rate and reducing tumor volume. While the safety profile included common adverse events like vomiting and rash, it was deemed acceptable given the unmet medical need. The report details the drug's pharmaceutical development, pharmacokinetics, toxicology, and non-clinical/clinical efficacy, leading to a conditional marketing authorization with requirements for long-term follow-up and post-authorization safety studies.
Key findings — cited to the page
| Stomatitis (CTCAE grade 3 and Above) | 1 % | NF1 paediatric pool · p.134 |
| Vomiting (CTCAE grade 3 and Above) | 8 % | NF1 paediatric pool · p.134 |
| Diarrhoea (all CTCAE grades) | 77 % | NF1 paediatric pool · p.134 |
| Stomatitis (all CTCAE grades) | 38 % | NF1 paediatric pool · p.134 |
| Grade ≥3 dermatitis acneiform | 4.0 % | · p.133 |
| Vomiting (all CTCAE grades) | 82 % | NF1 paediatric pool · p.134 |
| Nausea (CTCAE grade 3 and Above) | 1 % | NF1 paediatric pool · p.134 |
| patients with Grade ≥3 AEs | 62.0 % | SPRINT Phase II Stratum 1 · p.133 |
| Nausea (all CTCAE grades) | 73 % | NF1 paediatric pool · p.134 |
| Diarrhoea (CTCAE grade 3 and Above) | 15 % | NF1 paediatric pool · p.134 |
| Grade ≥3 dermatitis acneiform | 6.0 % | · p.133 |
| Rash (all CTCAE grades) | 80 % | NF1 paediatric pool · p.134 |
Source: AstraZeneca PLC ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →