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.

In response to NICE's final guidance, Pfizer said in a statement that it is "extremely disappointed that the NICE FAD does not recommend Vyndaqel for reimbursement".

"As the only authorised treatment for the condition, Pfizer strongly believes that [Vyndaqel’s] routine reimbursement will serve an important unmet need for patients in UK. NICE also chose to not uphold the appeal points submitted by Cardiomyopathy UK and the British Society for Heart Failure.

"We view the value assessments and clinical conclusions detailed in the report as out of step with consensus from clinical experts in this area. Pfizer has worked closely with NHS England and NICE throughout this submission. We remain steadfast in our commitment to finding a solution to make this important medicine available to eligible UK patients as quickly as possible," the company added.