Akcea Therapeutics has managed to convince NICE, the health technology appraisal (HTA) institute for England and Wales, to recommend its ultra-rare disease treatment, Tegsedi (inotersen), for hereditary transthyretin-related amyloidosis (hATTR) for use on the National Health Service. Following initial negative recommendations from NICE, the company submitted new evidence, economic modeling, and crucially, an “improved commercial arrangement.”
Akcea hopes other HTA and reimbursement agencies across Europe will follow “NICE’s lead in making inotersen available as a treatment...








