RARE Daily

NICE Gives Akcea Nod for Tegsedi to Treat Ultra-Rare hAATR

April 16, 2019

Marie Daghlian

The National Institute for Health and Care Excellence, the United Kingdom’s drug price watchdog, has recommended Akcea Therapeutics’ Tegsedi as an option for the treatment of stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin-related amyloidosis, an ultra-rare progressive disease.

The positive recommendation comes after the biotech offered a confidential commercial agreement that included a lower price for the treatment.

Hereditary transthyretin-related amyloidosis (hATTR) is caused by an inherited mutation in the gene that affects the function of the transthyretin (TTR) protein, causing the liver to produce abnormal TTR protein that accumulates as in various body systems including the autonomic nervous system, peripheral nerves, heart, gastrointestinal system, eyes, and central nervous system. NICE estimates that about 150 people have hATTR in the United Kingdom. 

Inotersen is a novel, first-in-class 2’-O-2- methoxyethyl phosphorothioate antisense oligonucleotide that inhibits production of TTR in adults with hATTR amyloidosis. NICE had initially rejected Akcea’s treatment in December but reconsidered after Akcea offered the agency an improved commercial agreement.  

The NICE recommendation is a boost for Akcea, because rival Alnylam Pharmaceuticals’ Onpattro had been seen as a slightly more effective treatment based on clinical trial data. NICE also rejected Onpattro in December. Akcea quickly offered a discount to gain approval.

Tegsedi is self-administered once a week by subcutaneous injection while Onpattro is administered once every three weeks. Both drugs face competition from Pfizer’s Tafadamis, available for years in Europe as a treatment for polyneuropathy and set to gain approval in the United States and Europe to treat hATTR.

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