Christmas came a day early this week for Aegerion Pharmaceuticals (Cambridge, MA), winning FDA approval for Juxtapid (lomitapide), a small-molecule treatment for homozygous familial hypercholesterolemia (HoFH), a rare disease. FDA granted Aegerion orphan-drug status in 2011; company documents indicate that there are several thousand potential patients for the condition, which is generally resistant to other cholesterol treatments, and currently requires patients to undergo apheresis (a type of dialysis) at an estimated cost of $150,000 per year. The once-a-day capsule is also expected by Aegerion to win approval in Europe this year.
Canaccord Genuity, a New York investment firm, issued a “buy” rating at the announcement, noting that company management expects to price the drug at $200,000-$300,000 per year. It also called the REMS (risk evaluation and mitigation strategies) requirement that FDA imposed as “benign.” Juxtapid comes with a boxed warning for hepatic toxicity (liver damage) in some patients. The REMS program will require physician and pharmacist certification, and for physicians’ participation in a training session and completion of a pre-authorization before treatment. Aegerion is also obliged to conduct three post-marketing trials, including developing long-term patient registry.
Lomitapide has had a long journey to commercialization. It was originally developed at Bristol Myers Squibb in the mid-1990s as a competitor to statins; BMS gave the rights to the drug to the University of Pennsylvania in 2003, and Aegerion acquired them in 2006. At least 23 Phase I-III trials have been performed. Aegerion itself went public in 2010; this is its first commercial product.