Pfizer has obtained the European Commission (EC) approval for VYNDAQEL (tafamidis), a once-daily 61 mg oral capsule, for hereditary transthyretin amyloidosis in cardiomyopathy patients (ATTR-CM).

The pharmaceutical firm said that its VYNDAQEL marks the first and only approved treatment for ATTR-CM in the European Union (EU). Before the EC approval, the treatment options for ATTR-CM patients were limited to symptom management, and heart transplant, in rare cases.

Pfizer rare disease global president Paul Levesque said: “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.

“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.”

VYNDAQEL is an oral transthyretin stabiliser that selectively binds to transthyretin

ATTR-CM is a rare, underdiagnosed and lethal disease characterised by the accumulation of abnormal deposits of a misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure.

The median life expectancy of patients with diagnosed ATTR-CM is approximately two to 3.5 years, subject to the disorder sub-type.

Pfizer said that the results from Phase 3 ATTR-ACT study, the global, double-blind, randomised, placebo-controlled clinical trial, which evaluated a pharmacologic therapy for ATTR-CM, has supported the EC approval of VYNDAQEL.

In the study, VYNDAQEL has shown a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations than placebo.

In addition, VYNDAQEL has shown consistent treatment effects compared to placebo on functional capacity and health status and reduced the decline in performance and health status.

French Referral Centres for Cardiac Amyloidosis coordinator Thibaud Damy said: “Before today, the European transthyretin amyloidosis community had a dire need for new therapeutic options that can improve outcomes for patients with cardiomyopathy.

“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.”