Pfizer Inc. — Vyndaqel EMA EPAR public assessment report (EMEA/H/C/002294, 2011)
Pfizer Inc. · 2011 · EMA EPAR public assessment report · company profile →
Report summary
This EMA report details the assessment of Vyndaqel (tafamidis meglumine), an orphan drug for transthyretin amyloidosis with polyneuropathy, which received a positive marketing opinion in 2011. Tafamidis stabilizes the TTR tetramer. Preclinical studies showed high bioavailability, widespread distribution, and dose-dependent toxicities, including reproductive concerns. Clinical trials, particularly the pivotal Fx-005 study, demonstrated efficacy in slowing disease progression and TTR stabilization, despite initial primary endpoint failures. Safety data revealed common adverse events like UTIs and GI issues. The CHMP granted authorization under exceptional circumstances, acknowledging benefit-risk for V30M and extrapolated non-V30M patients, while requiring further data and monitoring for potential risks like hepatotoxicity.
Key findings — cited to the page
| NOAEL (reproduction, rats) | ≥ 30 mg/kg/day | · p.20 |
| NOAEL (reproduction, rats) | ≥ 45 mg/kg/day | · p.20 |
| NOAEL (reproduction, rabbits) | 2 mg/kg/day | · p.20 |
| Mortality (rats) | 2 F | · p.18 |
| Marketing Authorisation application submission date | 28 July 2010 | Vyndaqel · EU · p.5 |
| Orphan medicinal product designation date | 28 August 2006 | Vyndaqel · EU · p.5 |
| Mortality (rats) | 3 F | · p.18 |
| CHMP positive opinion date | 21 July 2011 | Vyndaqel · EU · p.6 |
| Estimated patient population with ATTR-PN | 2,700 to 3,500 patients | Europe · p.7 |
| Estimated patient population with ATTR-PN | 5,000 to 10,000 patients | Global · p.7 |
| Liver transplants reported to FAPWTR since 1995 | more than 1,500 | · p.7 |
| Orphan medicinal product prevalence | less than 0.1 per 10,000 EU population | Vyndaqel · EU · p.5 |
Source: Pfizer Inc. ↗ · 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 Neurology library →