NICE recommends Akcea's Tegsedi for rare, life-threatening progressive disease

Akcea Therapeutics has shared the news that Tegsedi (inotersen), its antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production, has been recommended by NICE for use on the NHS in the treatment of stage 1 or stage 2 polyneuropathy with hereditary transthyretin amyloidosis (hATTR).

hATTR is a rare condition caused by abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in the body’s tissues and organs. It progresses rapidly and can be fatal, with a median survival of just 4.7 years from diagnosis.    

"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. 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."

Phase 3 data submitted in support of the drug showed that use of Tegsedi led to "an improved course of neurological disease" compared to placebo over 15 months of treatment, meeting both of the trial’s primary endpoints. This was confirmed by a "significant change from baseline" compared to placebo in neuropathy and quality of life measures according to both the modified Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN).

However, NICE did also note that despite this, the long-term benefits of the drug could not be confirmed.

"This is a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date," commented 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."

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