The National Institute for Health and Care Excellence (NICE) has issue a positive recommendation for Tegsedi (inotersen) for treating stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
This decision will allow patients in England with this rare, inherited, severely debilitating and fatal disease to access the treatment on the NHS.
“This is a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date,” said Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association.
“There is a critical need for innovative new therapies for people across the UK living with this debilitating disease. We hope inotersen will be available to patients in the UK very soon.”
Inotersen is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production. It is the first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.
NICE has made the final recommendations on how inotersen should be used in the NHS. The NICE decision was based on clinical trial evidence that shows inotersen slows progression of the disease considerably, although its long-term benefits are uncertain.
The recommendation is expected to form the basis for NICE’s final Technology Appraisal Guidance (TAG), the final process step before the treatment is available on the NHS in England.
Once the final guidance is published, the NHS mandate requires that inotersen is available for routine use within 90 days.
“We are delighted with this news that patients can access inotersen in England,” commented Dr. Richard A. Jones, SVP Head of Europe for Akcea Therapeutics.
“hATTR amyloidosis is a debilitating disease that, to date, has had limited treatment options in the UK.”
The decision is based on evidence from the NEURO-TTR trial, a Phase 3 randomised placebo-controlled study evaluating inotersen compared to placebo.