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Canada’s drug-pricing regulator has levied the largest fine in its history against an Irish pharmaceutical company that charged an “excessive” price in Canada for a drug that treats an ultrarare genetic disorder.

The Patented Medicine Prices Review Board, or PMPRB, announced Tuesday that it had ordered Horizon Pharma to pay Ottawa more than $22-million. The board also ordered the company to cut the price of its drug, Procysbi, in half.

Procysbi is the brand name of a delayed-release version of a drug called cysteamine bitartrate that treats cystinosis, an inherited metabolic disorder believed to afflict about 100 Canadians. The drug prevents cystine, an amino acid, from accumulating in patients’ bodies and causing lethal organ damage, beginning with the kidneys.

In a decision issued Sept. 1, a PMPRB panel found that when Horizon began selling Procysbi in Canada in 2017, it did so with a sticker price that topped out at $226,665 a year for the heaviest patients.

That price might have been acceptable to the regulator if Procysbi were a breakthrough drug for a fatal disease with no other treatments. But for decades, Canadian cystinosis patients had access to an older version of cysteamine bitartrate through Health Canada’s Special Access Programme, which allows unapproved drugs for life-threatening diseases into the country when there are no alternatives available.

No company has applied to market the older version, called Cystagon, in Canada because it would be hard to turn a profit on a drug that sells for a small fraction of Procysbi’s price. The PMPRB panel heard testimony that Cystagon’s maximum price in Canada ranged from as little as $4,190.56 a year in Newfoundland and Labrador to as much as $21,133.50 in British Columbia in 2018.

The only difference between the two versions is that Procysbi has a patented coating that allows for slower absorption, meaning it can be taken every 12 hours instead of every six. The older drug’s more frequent dosing schedule forced patients to get up in the middle of the night to take their pills.

The panel found that Procysbi’s dosing schedule made it a “moderate” improvement over Cystagon, rather than a full-fledged breakthrough. That led the panel to conclude that Horizon should cut the price of its drug in half, rather than slashing it to meet the price of Cystagon.

Horizon said it won’t fight the panel’s decision or the $22-million fine in court. “We continue to firmly stand behind the value of our innovative rare disease medicine and the real-world value it provides for a very small, rare disease population,” Horizon spokesperson Amanda Phraner said in a statement.

The PMPRB’s ruling against Horizon is a departure for a regulator that usually settles allegations of excessive pricing through voluntary compliance orders. The federal Liberals promised five years ago to overhaul the PMPRB to lower Canada’s drug prices, but the government scaled back the reforms in the spring after the pharmaceutical lobby fought the changes on Parliament Hill and in court.