Draft NICE guidance does not recommend Onpattro for hereditary transthyretin-related amyliodsis.- Alnylam Pharmaceuticals.

The National Institute for Health and Care Excellence has issued draft guidelines rejecting NHS funding for Onpattro (patisiran), from Alnylam Pharmaceuticals, to treat the inherited condition hereditary transthyretin-related amyliodsis. Preliminary highly specialised technologies guidance concluded that the therapy offers short term benefits for people with the condition by slowing disease progression and improving quality of life.

However, it is uncertain whether these benefits are maintained in the longer-term. As a result cost-effectiveness estimates are much higher than the range that can be considered an appropriate use of NHS resources for highly specialised services.

Comment: Onpattro is based on Nobel Prize-winning science and is the first-ever RNA interference (RNAi) therapeutic to be approved in the European Union. The EC decision was based on the evaluation of the effects of patisiran in hATTR amyloidosis patients with polyneuropathy and its safety profile as demonstrated in the APOLLO Phase III study.