NICE releases draft guidance for patisiran for hATTR amyloidosis

NICE have released draft guidance for patisiran for adults living with hATTR amyloidosis who have stage 1 or 2 polyneuropathy.

NICE have released draft guidance for patisiran for adults living with hATTR amyloidosis who have stage 1 or 2 polyneuropathy.

As part of a Highly-Specialised Technology (HST) appraisal, the National Institute for Health and Care Excellence (NICE) has issued initial draft guidance for consultation regarding NHS use of the first-in-class RNA interference (RNAi) therapeutic, ONPATTRO™ (patisiran). Patisiran is used to treat adults living with hATTR amyloidosis who have stage 1 or 2 polyneuropathy. hATTR amyloidosis is a rare, life-threatening disease caused by abnormal deposition and accumulation of proteins in the tissues of the body.

At this draft stage, NICE is not yet able to recommend patisiran for routine NHS use. However, it was also noted by the Committee that patisiran is likely to provide “considerable clinical benefits to people with hATTR amyloidosis”, which it noted is an “exceptionally rare condition with a serious and substantial effect on the quality of life of patients, and their families and carers”.

Patisiran was granted marketing authorisation across the EU in August 2018, becoming the first-ever RNAi therapeutic to be approved for use in the UK. The MHRA also granted early access to patisiran for hATTR amyloidosis. It is based on Nobel Prize-winning science and is designed to target and silence specific messenger RNA, potentially blocking the production of a protein, called transthyretin (TTR), before it is made. By blocking the production of this protein in the liver, patisiran reduces its harmful accumulation in the body’s tissues – potentially halting or slowing the progression of this disease.

NICE has also conclude that inotersen (Tegsedi, Akcea Therapeutics) offered benefits for people with the condition in the short term by slowing progression of the disease and improving quality of life.

Commenting on the draft guidance, Brendan Martin, General Manager of Alnylam UK & Ireland said: “Patisiran is breaking new ground, offering eligible people with hATTR amyloidosis a new treatment option with the potential to help improve debilitating neuropathy symptoms.

“From the outset, both Alnylam and NICE recognised that the novel, highly-specialised nature of patisiran could make it challenging to assess its full value. This is why a HST was deemed the most appropriate appraisal process. 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.”