Esteve S A — Caprelsa EMA EPAR public assessment report (EMEA/H/C/002315, 2012)
Esteve S A · 2012 · EMA EPAR public assessment report
Report summary
The EMA's public assessment report for Caprelsa (vandetanib) details its conditional marketing authorization for aggressive and symptomatic medullary thyroid cancer (MTC), addressing an unmet medical need. Manufacturing processes are validated, and extensive non-clinical studies confirm vandetanib's inhibitory activity against key kinases and broad antitumor effects, though potential safety concerns like QT prolongation and reproductive toxicity are noted. Clinical trials, particularly the Phase III Study 58, demonstrated significant improvements in progression-free survival and objective response rates in MTC patients, especially those with RET mutations. While overall survival showed no significant difference due to crossover, the drug's efficacy led to approval. Safety concerns, including QTc prolongation, PRES, and renal toxicity, necessitate strict risk minimization strategies, ongoing pharmacovigilance, and educational materials. The benefit-risk balance is considered positive.
Key findings — cited to the page
| Daphnia sp. Reproduction Test (OECD 211) NOEC | 300 µg/L | · p.21 |
| Vandetanib 100 mg tablets batches manufactured at production scale | 3 | · p.14 |
| Increased oestrus cycle irregularity in female fertility study at 10 or 25 mg/kg/day | 10 or 25 mg/kg/day | · p.19 |
| Collembola, Reproduction Test (ISO 11267) NOEC | 10 mg/kg | · p.21 |
| Vandetanib 300 mg tablets batches manufactured at production scale | 3 | · p.14 |
| Total number of patients randomized | 331 | Study 58 · p.38 |
| Number of patients randomized to placebo arm | 100 | Study 58 · p.38 |
| PFS events in vandetanib 300mg arm | 73 | Study 58 · p.39 |
| Percentage of patients with RET mutation negative status | 2.4 % | Study 58 · p.38 |
| Patients with RET mutation negative status | 8 | Study 58 · p.38 |
| Patients with unknown RET mutation status | 136 | Study 58 · p.38 |
| Number of patients randomized to vandetanib 300mg arm | 231 | Study 58 · p.38 |
Source: Esteve S A ↗ · 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 →