The approval from the European Commission (EC) makes VYNDAQEL the first and only treatment for patients with ATTR-CM in the European Union (EU).
Considered to be a rare, underdiagnosed and life-threatening disease, ATTR-CM is characterized by the accumulation of abnormal deposits of misfolded protein called amyloid in the heart. The disease is defined by restrictive cardiomyopathy and progressive heart failure.
The Pfizer drug is also the only EC-approved medicine that has been proven to bring down mortality and frequency of cardiovascular-related hospitalisations in adults having wild-type or hereditary ATTR-CM.
VYNDAQEL’s approval by the EC was driven by the data from the phase 3 ATTR-ACT trial featuring patients with wild-type or hereditary ATTR-CM. The late-stage clinical study compared patients who were subjected to an oral daily dose of 20mg or 80mg of tafamidis meglumine compared to those who were given placebo.
In the primary analysis of the ATTR-ACT trial, VYNDAQEL delivered a substantial reduction in the hierarchical combination of all-cause mortality and frequency of hospitalisations caused by cardiovascular events compared to placebo over a 30-month period.
Pfizer rare disease global president Paul Levesque said: “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.”
The EC had approved the tafamidis meglumine 20mg capsule formulation of VYNDAQEL in 2011 for transthyretin amyloidosis in adults having stage 1 symptomatic polyneuropathy (ATTR-PN) for delaying peripheral neurologic impairment.
In May 2019, the US Food and Drug Administration (FDA) approved both tafamidis meglumine and tafamidis for their use in patients with ATTR-CM.
According to Pfizer, the two oral transthyretin stabilisers have been designed to bind selectively to transthyretin, thereby stabilising the tetramer of the transthyretin transport protein and slowing the formation of amyloid that leads to the heart condition.