Vyndaqel tafamidis meglumine regulatory update
The German Institute for Quality and Efficiency in Healthcare (IQWiG) said Vyndaqel tafamidis from Pfizer plus best supportive care provides a "small degree" of additional benefit compared with best supportive care alone in the treatment of transthyretin (TTR) familial amyloid polyneuropathy (FAP). Best supportive care is the best individualized care to mitigate symptoms and improve quality of life. Vyndaqel has Orphan Drug designation in the EU.
In a preliminary benefit assessment, IQWiG said Vyndaqel slows progression of neurological damage. However, the agency did not find a statistically significant difference between Vyndaqel and best supportive care in other patient-relevant endpoints, including overall survival (OS), quality of life and side effects. ...