The National Institute of Health and Care Excellence (NICE) has issued draft highly specialised technologies guidance, now recommending Akcea Therapeutics’ Tegsedi (inotersen) to treat the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR).

The recommendation follows an improved commercial agreement for the drug from the company, which addresses the uncertainties in the company’s economic model, and is for patients with stage 1 or 2 polyneuropathy.

According to Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association, “This is a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date.”

“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.”

It’s an important decision as it will allow patients in England with this rare, inherited, severely debilitating and fatal disease to access the treatment on the NHS.

hATTR is 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.

“We are delighted with this news that patients can access inotersen in England,” commented Dr. Richard 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.

“We hope that other health technology assessment and reimbursement agencies across Europe will take NICE’s lead in making inotersen available as a treatment option for patients with this disease. This positive news further confirms Akcea’s commitment to advancing and making accessible transformative treatments for patients living with serious and rare diseases.”

The drug is the first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.