Genzyme and Aegerion’s Treatments for HoFH, Explained

April 16, 2013

In late December 2012 and January 2013, the FDA approved Genzyme’s Kynamro and Aegerion Pharmaceutical’s Juxtapid, two products that treat homozygous familial hypercholesterolemia (HoFH). These drugs were both granted orphan status, as HoFH affects about 3,000 people in the United States.

Although it is unusual for two products that treat the same orphan disease to come to market at the same time, competition between the two has been questioned. Genzyme’s Kynamro, which was released second, is priced at $176,000 per patient per year, while Aegerion’s is priced at $295,000.

Treatment Details

But let’s break down the cost, peering into their benefits, specifics, etc.

Q: How effective are these treatments?

A: Both aim to tame patients’ LDL cholesterol levels. Genzyme’s Kynamro dropped patients’ LDL-C levels 25 percent on average across four Phase III studies, while Aegerion’s Juxtapid saw around a 40 percent drop in LDL-C levels.

Q: What about delivery or frequency of drug taking?

A: Kynamro is a once-a-week shot; Juxtapid is a daily pill. With so many patients already taking as many as 17 pills a day, a single weekly injection may be more appealing to some patients. Both new drugs have roused safety concerns, and each carry Boxed Warnings of possible liver toxicity.

Before They Came to Market

Q: Did this alleged competition begin before these treatments came to market; what was the atmosphere between the two during Research & Development? Recruiting for clinical trials is notoriously difficult in the orphan space, and the amount of subjects is further limited when multiple companies are striving to develop products for the same indication.

A: Mike McCann, VP, global strategy marketing in Genzyme’s cardiovascular division says the two firms’ studies had some overlap, but Genzyme did not face added difficulty in recruitment. That is because Aegerion sought a different type of patient. Juxtapid had placebo-controlled trials for patients receiving LDL apheresis; Kynamro’s trials did not include patients on the older treatment.

A CEO’s Perspective

Marc Beer, CEO of Aegerion Pharmaceuticals, said it best, “We don’t view it as a race against competition, but rather a race against this terrible disease…The patient is waiting…There’s still much to be done to achieve our commitment of delivering this important therapy to HoFH patients.”

Story compiled by Katie Mastro.

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