
Akcea’s Tegsedi secures NICE approval for Stage 1 or 2 polyneuropathy in hATTR
pharmafile | May 23, 2019 | News story | Sales and Marketing | Akcea Therapeutics, NHS, NICE, Tegsedi, UK, pharma
Akcea Therapeutics’ Tegsedi (inotersen) has received recommendation from NICE, the company has revealed, meaning the therapy will now be made available to NHS patients in England and Wales for the treatment of Stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
hATTR is a serious condition caused by “the abnormal formation of of the TTR protein and aggregation of TTR amyloid deposits” in the body’s tissues, including in the heart, peripheral nerves and intestinal tract. As the disease progresses, peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy often manifest, with patients living for only a median of 4.7 years following diagnosis.
As per the NHS mandate, the therapy will now be available to patients via the health service in England within 90 days of the recommendation, while Welsh patients will have access earlier due to a commitment by the NHS in Wales to having funding available earlier.
“This is a great milestone for inotersen and marks the successful conclusion of this health technology assessment for inotersen,” commented Dr Richard A Jones, SVP Head of Europe for Akcea Therapeutics. “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.”
Matt Fellows
Related Content

NIHR chief scientific adviser visits Huddersfield health innovation campus
Professor Lucy Chappell, Chief Scientific Adviser to the Department of Health and Social Care and …

Pharma sees climate disruption as growing supply-chain risk
Nine in ten pharmaceutical companies across the UK and G7 countries expect climate-related disruption to …

Five Facts about NHS and primary care
1 Primary care is the front door to the NHS, but it extends well beyond …





