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UK price watchdog rejects 2 rare genetic disease drugs from Akcea, Alnylam

The U.K. National Institute for Health and Care Excellence did not recommend Akcea Therapeutics Inc.'s Tegsedi and Alnylam Pharmaceuticals Inc.'s Onpattro, both used to treat a rare hereditary disorder.

Tegsedi and Onpattro are lifelong treatments intended for polyneuropathy, or nerve damage outside the brain and central nervous system, in adults with hereditary transthyretin-related amyloidosis.

Hereditary transthyretin-related amyloidosis, or hATTR, is a rare, genetic, progressive disease characterized by the formation of the protein called amyloid in the peripheral nerves, heart or gastrointestinal tract. According to NICE, about 150 people in England are affected by the condition.

In two separate Dec. 12 draft guidances, the drug price watchdog said both drugs offer benefits — delay in disease progression and quality of life improvement — that are deemed short term in nature, and it is still uncertain if they will be maintained in the long run.

NICE also raised concerns related to the economic modeling for both Tegsedi and Onpattro. The U.K. health agency cited uncertainties in Tegsedi's utility values, number of carers, mortality and stopping of the treatment. For Onpattro, NICE said the drug covers some important aspects of hATTR but not the other more subjective symptoms.

The regulator also noted that both drugs are not cost-effective as their prices are much higher than what is considered acceptable for highly specialized technologies.

Tegsedi, or inotersen, works by reducing the production of the transthyretin protein through a once-weekly injection that patients can administer themselves through the skin. The injection developed by Akcea and Ionis Pharmaceuticals Inc. costs £5,920 per 284-milligram treatment on a weekly dose.

Onpattro, or patisiran, is an RNA-based treatment that was also designed to prevent the production of transthyretin by the liver. Alnylam's drug is administered through an intravenous infusion every three weeks at a 0.3-milligram per kilogram dosage. The treatment costs £7,176.45 for each 10-milligram vial.

Tegsedi and Onpattro are already approved by the U.S. Food and Drug Administration to treat polyneuropathy caused by hATTR in adults.