The path for Ionis’ Tegsedi in the U.K. looked rocky after a preliminary "no" last year from England's drug-price watchdog. Fortunately for the drugmaker, that decision was reversed.

Tuesday, the National Institute for Health and Care Excellence (NICE) released final guidance recommending Tegsedi’s placement on England’s National Health Service for treatment of nerve damage from hereditary transthyretin-mediated amyloidosis (ATTR), an ultra-rare disease that affects about 150 U.K. citizens, NICE said in an email.

NICE reversed its early guidance from October 2018 not recommending Tegsedi, which is in a head-to-head battle with Alnylam Pharmceuticals' Onpattro, saying the drug’s cost efficiency over palliative care had improved after the drugmaker agreed to a steeper discount for its £5,925 ($7,737)-per-week treatment.

In its final guidance, NICE said the drug slowed the progression of ATTR, which causes the production of abnormal transthyretin protein in the liver, but had unclear long-term effects on top of an "uncertain" economic model. However, the price break Ionis served up was enough for a vote of confidence.

"Some assumptions in the economic modelling are also uncertain, particularly around the utility values and the healthcare costs," the guidance said. "Despite the uncertainties, (Tegsedi) is likely to provide important clinical benefits for people with (ATTR) and value for money within the context of a highly specialized service."

Prior to the introduction of Tegsedi and Onpattro, the only treatment for ATTR was supportive care.

While Tegsedi has had a bumpy ride into the English market, rival Onpattro’s path hasn’t been a cakewalk, either. Alnylam’s entry into the field received a similar "no" from NICE in October 2018 after the watchdog said the drug’s cost efficiency ratio per quality-adjusted life year gained—a metric NICE uses to establish cost-effectiveness—wasn’t up to snuff.

That knock in the U.K. compounded a rough first quarter of sales for Onpattro, which received FDA approval in August 2018. According to the company, Onpattro pulled in just $460,000 in revenue between its approval month and the end of the third quarter.

Tegsedi received its FDA approval in October 2018.

The two companies could soon see a third challenger enter the ring as Pfizer’s tafamidis seeks its own FDA approval to treat ATTR. In August, the heavyweight drugmaker announced positive phase 3 trial data for ATTR cardiomyopathy—a different form of the disease, but one in which existing competitors hope to eventually compete.

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