The UK subsidiary of Akcea Therapeutics today announced that the National Institute for Health and Care Excellence (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 National Health Service (NHS).
“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,” he noted.
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.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze