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Europe approves first treatment for transthyretin amyloidosis in cardiomyopathy patients: Pfizer's Vyndaqel

Published on 19/02/20 at 12:17pm

The European Commission (EC) has awarded marketing approval to Pfizer’s Vyndaqel (tafamidis), it has emerged, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult cardiomyopathy patients (ATTR-CM).

The decision makes it the first and only treatment for ATTR-CM available to patients in the European Union; previously available treatments were limited to the management of symptoms or transplant of the heart and liver in rare cases.

Data supporting the decision showed that daily, oral Vyndaqel 20mg or 80mg “had significant and consistent treatment effects compared to placebo on functional capacity and health status”, effects first identified after six months of treatment and maintained through to 30 months.

A rare and underdiagnosed condition, ATTR-CM is characterised by accumulation in the heart of the misfolded protein amyloid in the heart, causing progressive heart failure and restrictive cardiomyopathy, eventually leading to death between two and 3.5 years, depending on disease sub-type.

“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 of Pfizer Rare Disease. “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.”

Matt Fellows

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