Tegsedi™▼ to be available on the NHS for hATTR amyloidosis

Dependence on prescription medicines uncovered in Public Health England review

NICE issues positive recommendation for inotersen to treat adult patients with stage 1 or stage 2 polyneuropathy with hATTR amyloidosis. 

Akcea Therapeutics UK Ltd., has announced that NICE has issued a positive Final Evaluation Document (FED) for Tegsedi™ (inotersen) for the treatment of stage 1 or stage 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.

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.The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.

hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years from diagnosis.

“This is a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date”

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.

By publishing a FED, 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 in the FED 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.

The decision is based on evidence from the NEURO-TTR trial, a Phase 3 randomised placebo-controlled study evaluating inotersen compared to placebo. It demonstrated that patients taking inotersen had an improved course of neurological disease over the 15-month study period confirmed by the significant change from baseline compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) which were the two primary endpoints of the study.

Inotersen is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified safety risks.

In July 2018 Akcea received European Commission approval of Tegsedi™ ▼

Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association commented: “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.”

Dr. Richard A. Jones, SVP Head of Europe for Akcea Therapeutics said, “We are delighted with this news that patients can access inotersen in England. 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.”

In December 2018 NICE released draft guidance for patisiran for hATTR amyloidosis