The National Institute for Health and Care Excellence has issued draft guidelines rejecting NHS funding for two therapies that treat the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR).

Preliminary highly specialised technologies guidance on Akcea Therapeutics’ Tegsedi (inotersen) and Alnylam Pharmaceuticals’ Onpattro (patisiran) conclude that both therapies offer benefits for people with the condition in the short term by slowing progression of the disease and improving quality of life.

However, it is “uncertain” whether these benefits are maintained in the longer-term, and there were further in the economic modelling for both treatments, NICE said.

Consequently, cost-effectiveness estimates for both were “much higher” than the range that can be considered an appropriate use of NHS resources for highly specialised services, the Institute stressed.

hATTR is an ultra-rare (in England it is thought to affect around 150 people), inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene, which cause abnormal amyloid proteins to accumulate and damage body organs and tissue, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.

There are no therapies available to treat the underlying cause of hATTR, so current treatments are limited and mainly focus on symptom relief and supportive care, including pain management, nutritional and mobility support and mitigation of the effects of the disease on other organs.

Onpattro, which works by stopping production of transthyretin by the liver, was cleared for use in Europe back in August, following approval of Tegsedi , which suppresses transthyretin production, in July.

“Patisiran is breaking new ground, offering eligible people with hATTR amyloidosis a new treatment option with the potential to help improve debilitating neuropathy symptoms,” said Brendan Martin, general manager of Alnylam UK & Ireland.

“While we believe our current proposal demonstrates that patisiran represents real value, we welcome the recognition by NICE that this medicine provides ‘considerable clinical benefits’ and we look forward to continuing discussions about how we can make it available to patients in the UK.

"Our focus now will be on addressing the immediate questions posed by the Committee and on working towards securing a positive, sustainable agreement that represents value for the NHS and that would help transform the lives of people living with this terrible disease.”