The National Institute for Health and Care Excellence (NICE) in the UK has issued positive recommendation for Akcea Therapeutics ’ Tegsedi (inotersen) to treat stage 1 or stage 2 polyneuropathy in hereditary transthyretin amyloidosis (hATTR) patients.

NICE published a final evaluation document (FED) that is expected to support its final technology appraisal guidance (TAG), which will provide patients in England with access to the drug on the National Health Service (NHS).

Akcea Therapeutics will be required to make inotersen available for routine use on the NHS within 90 days of the TAG publication.

hATTR amyloidosis is a progressive condition developed due to abnormal human transthyretin (TTR) protein production and accumulation of TTR amyloid deposits throughout the body.

The disorder can cause cardiomyopathy, autonomic dysfunction and polyneuropathy, alongside a catalogue of other symptoms. Patients have a median survival of 4.7 years from diagnosis.

Discovered and developed by Ionis Pharmaceuticals , inotersen is an antisense oligonucleotide (ASO) designed to inhibit TTR production. It is said to be the first subcutaneous RNA-targeting drug which fixes onto the TTR protein formation.

“hATTR amyloidosis is a debilitating disease that has had limited treatment options in the UK.”

Akcea Therapeutics Europe senior vice-president head Richard Jones said: “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.”

NICE decision is based on data from the randomised, placebo-controlled Phase III NEURO-TTR clinical trial performed in a total of 172 subjects to compare inotersen with placebo.

Results demonstrated that inotersen slowed progression of the disease, with patients experiencing an improved course of the disease over the trial duration of 15 months.

The improvement was confirmed by significant change from baseline in measures of neuropathy and quality of life, the primary endpoints of the trial, compared to placebo.

Based on this data, inotersen also secured the European marketing authorisation.

Long-term benefits of the drug, which is linked to risk of thrombocytopenia – low blood plate count – and glomerulonephritis – kidney damage – are yet to be determined.