Akcea Therapeutics UK has announced that the National Institute for Health and Care Excellence (NICE) has published a final guideline for its nerve damage drug, Tegsedi (inotersen).

The Highly Specialised Technologies (HST) Guidance is for the treatment of stage I or II polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

It follows the positive Final Evaluation Document (FED) published last month recommending that patients in England with this rare, inherited, severely debilitating and fatal disease, can access the treatment on the NHS.

The drug is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production, and is the first and only subcutaneous RNA-targeting drug designed to reduce the production of TTR protein.

This HST Guidance is the final recommendation from NICE regarding how Tegsedi should be used in the NHS in England. Now that the final guidance has been published, the NHS mandate requires that the drug is available for routine use within 90 days for patients in England.

The achievement is a “great milestone” for Tegsedi, says Dr. Richard A. Jones, SVP head of Europe for Akcea Therapeutics, and “marks the successful conclusion of this health technology assessment for inotersen.”

He continued, “Patients in the UK with hATTR amyloidosis have to date had very limited treatment options to date, so this news will be well received among the amyloidosis community. Akcea are committed to advancing and making accessible transformative treatments for patients living with serious and rare diseases.”

hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract.