Vyndaqel becomes first treatment in the EU for ATTR-CM

Following a positive recommendation by a European Medicines Agency advisory committee in December for an extended indication, the European Commission (EC) has now granted final approval for Vyndaqel (tafamidis), a once-daily 61mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Vyndaqel, developed and marketed by US pharma giant Pfizer (NYSE: PFE), is the first and only treatment approved in the European Union for patients with ATTR-CM. Prior to this approval, treatment options for patients with ATTR-CM were restricted to symptom management, and, in rare cases, heart (or heart and liver) transplant.

Vyndaqel was approved for the treatment of the rare and fatal neurodegenerative hereditary disease transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy in 2011.