Novartis AG — Rydapt EMA EPAR public assessment report (EMEA/H/C/004095, 2017)
Novartis AG · 2017 · EMA EPAR public assessment report · company profile →
Report summary
The EMA granted marketing authorization for Rydapt (midostaurin) on July 20, 2017, for newly diagnosed FLT3 mutation-positive acute myeloid leukaemia (AML) and advanced systemic mastocytosis (SM). Rydapt, a new therapeutic agent, targets FLT3 and KIT. Pivotal studies demonstrated significant efficacy: in FLT3-mutated AML, midostaurin improved overall survival and event-free survival compared to placebo. For advanced SM, it achieved notable overall response rates in a single-arm study. Pharmacokinetic analysis revealed time-dependent kinetics and CYP3A4 metabolism, with strong inhibitors/inducers significantly impacting exposure. Key safety concerns include myelosuppression, gastrointestinal events, and cardiac toxicities, particularly QT prolongation. A risk management plan addresses these identified and potential risks.
Key findings — cited to the page
| Diarrhoea (all grades) | 74.8 % | All sites · p.179 |
| Diarrhoea (all grades) | 73.6 % | All sites · p.179 |
| Lipase increased (Grade 3/4 laboratory abnormality) | 17.6 % | · p.187 |
| Hyperglycaemia (Grade 3/4 laboratory abnormality) | 19 % | · p.187 |
| grade 3/4 hypotension (midostaurin vs placebo) | 10 vs 1 patients | · p.199 |
| Diarrhoea (Grade 3/4) | 14.5 % | All sites · p.179 |
| Absolute lymphocyte count decreased (Grade 3/4 laboratory abnormality) | 45.8 % | · p.187 |
| Grade 3-4 nausea (midostaurin) | 6.0 % | D2201 · p.203 |
| grade 3/4 febrile neutropenia | 44 % | · p.199 |
| ANC decreased (Grade 3/4 laboratory abnormality) | 26.8 % | · p.187 |
| grade 3/4 exfoliative dermatitis (midostaurin vs placebo) | 10 vs 1 patients | · p.199 |
| Diarrhoea (Grade 3/4) | 16.9 % | All sites · p.179 |
Source: Novartis AG ↗ · 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 →