Hyperion’s Pinpoint Mission Pays Off with Drug Approval

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acquire the older drug from Medicis, which was recently acquired by the Montreal, Canada-based Valeant (NYSE: VRX). Medicis has been focusing on drugs that have a cosmetic effect, such as Restylane, a gel injected into the skin to change the appearance of wrinkles.

Hyperion is following in the path of other orphan drug makers such as Genzyme and Biomarin Pharmaceuticals (NASDAQ: BMRN), which have shown that serving very small patient populations can be a profitable business. Health plans will reimburse for expensive drugs that treat life-threatening conditions, especially when the treatments help avoid hospitalizations.

Santel says Hyperion will develop ways to help patients get access to the drug if they can’t afford it.

“I think we’re going to do the right thing as corporate citizens,” he says.

Hyperion will probably seek a partner to market Ravicti outside the United States, Santel says.

The company has also completed a Phase 2 trial of the drug in a second indication, hepatic encephalopathy, a nervous disorder linked to liver damage or liver failure. The neurological symptoms, ranging from mild disorientation to coma, may result from toxins such as ammonia that the liver has failed to remove from the blood. The patient population for this use is much bigger. Hyperion estimates that about 140,000 people in the United States are known to suffer from hepatic encephalopathy, and about 1 million have cirrhosis, a scarring of the liver and poor liver function.

Hyperion expects to begin a Phase 3 trial in hepatic encephalopathy in 2014.

The company has raised about $160 million to date through its IPO and other fundraising rounds. Santel says the staff will have plenty to do as they prepare to start selling Hyperion’s first product, but the company suggests it may have even bigger ambitions.

“We always have our eyes open for other opportunities,” Santel says.

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