Pfizer’s Vyndaqel has been rejected by the UK National Institute for Health and Care Excellence (NICE) as a treatment for the rare heart condition transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

The final guidance publication comes following an appeal against earlier draft recommendations by Pfizer, Cardiomyopathy UK and the British Society for Heart Failure, NICE said in a statement.

In the UK, around 800 people live with ATTR-CM, a rare, under-diagnosed and life-threatening disease characterised by the buildup of abnormal deposits of misfolded protein called amyloid in the heart.

Once diagnosed, the median life expectancy in patients with ATTR-CM, dependent on sub-type, is approximately two to 3.5 years.

NICE said that cost-effectiveness estimates for Vyndaqel are higher than what would normally be considered an acceptable use of NHS resources.

The agency added that there isn’t enough evidence that a recommendation for Pfizer’s drug would reduce delays in diagnosis, with further uncertainty regarding the long-term benefit of Vyndaqel once treatment is stopped.

NICE said that it ‘remains keen’ to ensure access to Vyndaqel for ATTR-CM patients, adding that it is willing to conduct a rapid review of the final guidance if NHS England and Pfizer can reach an agreement for a new value proposition which may make the drug cost-effective.

In February 2020, Vyndaqel became the first treatment approved in Europe for the treatment of ATTR-CM.

The European Commission based this approval on from the Phase III ATTR-ACT study, the first and only completed global, double-blind, randomised, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM.

In the study, the drug was shown to reduce the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo.