Daiichi Sankyo Company, Limited — Vanflyta EMA EPAR public assessment report (EMEA/H/C/005910, 2023)
Daiichi Sankyo Company, Limited · 2023 · EMA EPAR public assessment report · company profile →
Report summary
Daiichi Sankyo's Vanflyta (quizartinib) received a positive EMA opinion in September 2023 for newly diagnosed FLT3-ITD positive acute myeloid leukaemia (AML). This Type II FLT3 inhibitor demonstrated a statistically significant improvement in overall survival (median 31.9 vs. 15.1 months for placebo) in the Phase 3 Quantum-First study, despite higher early deaths and non-significant event-free survival. Non-clinical and clinical studies detailed its pharmacokinetics, high plasma protein binding, and primary faecal excretion. While effective, quizartinib is associated with increased adverse events, including neutropenia, QT prolongation, and infections, necessitating risk minimization strategies. Its development involved extensive quality control, non-clinical toxicology (bone marrow, lymphoid, liver, kidney, reproductive organ toxicities), and clinical efficacy assessments across various doses and patient populations.
Key findings — cited to the page
| Related Grade 3/4 AEs: Overall in quizartinib arm | 43% | · p.126 |
| Related Grade 3/4 AEs: Overall in placebo arm | 22.8% | · p.126 |
| Related Grade 3/4 AEs: Neutropenia in quizartinib arm | 15.5% | · p.126 |
| Related Grade 3/4 AEs: Neutropenia in placebo arm | 3.7% | · p.126 |
| NOAEL for general toxicity and reproduction in dams and for embryo-foetal development | 2 mg/kg/day | · p.29 |
| Orphan designation withdrawal date | 19 September 2023 | Vanflyta · EU · p.8 |
| Orphan medicinal product designation | EU/3/09/622 | Vanflyta · EU · p.8 |
| Incidence of FLT3-ITD mutations (20-59 years) | up to 35 % | · p.12 |
| Marketing authorisation positive opinion date | 14 September 2023 | Vanflyta · p.11 |
| 5-year survival rate for AML | 40 % | · p.12 |
| Incidence of AML | 2.5 to 6 per 100,000 people | Europe · p.12 |
| Subjects undergoing HSCT (60 mg group) | 42.1 % | 2689-CL-2004 · p.53 |
Source: Daiichi Sankyo Company, Limited ↗ · 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 →