European Commission Issues Approval of Pfizer’s Vyndaqel

The European Commission (EC) has approved Pfizer’s treatment for wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM), Vyndaqel (tafamidis), it was announced in a Feb. 18, 2020 press release.

“Until today, there were no approved medicines to treat patients with ATTR-CM in the EU. Today’s approval represents incredible progress for these patients and reflects our steadfast commitment to delivering breakthrough medicines to rare disease patients,” said Paul Levesque, global president, Pfizer Rare Disease, in the press release. “Additionally, with today’s milestone, Vyndaqel is now the first treatment to have two formulations approved in the EU to treat manifestations of transthyretin amyloidosis: one for cardiomyopathy, and one for stage 1 polyneuropathy.”

“Before today, the European transthyretin amyloidosis community had a dire need for new therapeutic options that can improve outcomes for patients with cardiomyopathy,” added Thibaud Damy, MD, coordinator of the French Referral Centers for Cardiac Amyloidosis and past president of the French Heart Failure and Cardiomyopathy group, French Society of Cardiology, in the press release. “Vyndaqel represents a major advance for patients, as it can significantly reduce all-cause mortality and the frequency of cardiovascular-related hospitalizations in patients with wild-type or hereditary ATTR-CM.”

Vyndaqel is a once-daily 61 mg oral capsule that has been found to significantly reduce the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations when compared to placebo in a Phase III clinical study. Additionally, the pharmacologic therapy was found to be well-tolerated and had significant and consistent treatment effects in the clinical study.

Source: Pfizer