Pfizer Inc. — Vyndaqel EMA EPAR variation assessment report (EMEA/H/C/002294, 2011)
Pfizer Inc. · 2011 · EMA EPAR variation assessment report · company profile →
Report summary
Pfizer sought to extend Vyndaqel's (tafamidis) marketing authorization to include a new 61 mg strength and a new indication for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The application involved updating the existing 20 mg formulation's product information and the Risk Management Plan. The CHMP granted the new indication, with the 61 mg dose bioequivalent to 80 mg tafamidis meglumine. Non-clinical and clinical pharmacology, including pharmacokinetics, pharmacodynamics, and toxicology, were assessed. Clinical trials demonstrated tafamidis's superior efficacy over placebo in reducing all-cause mortality, cardiovascular-related hospitalizations, and improving functional measures over 30 months. The safety profile was comparable to placebo, with some concerns regarding hepatotoxicity and thyroid dysfunction at higher doses. Despite some uncertainties regarding optimal dosing and benefits in specific patient subgroups, the CHMP concluded a positive benefit-risk balance, recommending the extension for both wild-type and hereditary ATTR-CM.
Key findings — cited to the page
| Prevalence of wild-type ATTR-CM in heart failure patients with preserved ejection fraction | 13% | · p.9 |
| Prevalence of wild-type ATTR-CM in patients undergoing transcatheter aortic valve replacement for severe aortic stenosis | 16% | · p.9 |
| Waiver number for product-specific waiver | EMEA-000884-PIP01-10 | · p.7 |
| LS mean change from Baseline in 6MWT at Month 30 (placebo) | -130.55 metres | Study B3461028 · p.42 |
| Prevalence of ATTR wild type (previously SSA) | approximately 3 in 10,000 people | EU · p.6 |
| EMA Decision number for product-specific waiver | P/147/2010 | · p.7 |
| Discontinuation due to SAEs for tafamidis 20 mg group | 17 % | B3461028 Cohort · p.71 |
| Date of positive opinion for marketing authorisation | 12 December 2019 | Vyndaqel · p.8 |
| Prevalence of hereditary ATTR-CM | below 0.1 in 10,000 people | EU · p.9 |
| Prevalence of wild-type ATTR | approximately 3 in 10,000 people | EU · p.9 |
| Prevalence of wild-type ATTR-CM in patients with presumed hypertrophic cardiomyopathy | 5% | · p.9 |
| Mean progression to death for ATTR-CM variants | 2 to 3 years | · p.9 |
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 →