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12:00 AM
 | 
Feb 04, 2013
 |  BioCentury  |  Strategy

Head to head in hoFH

Aegerion: Juxtapid data will drive use in hoFH despite price; Genzyme's Kynamro

Aegerion Pharmaceuticals Inc. thinks its data will support broad use of Juxtapid lomitapide to treat homozygous familial hypercholesterolemia, despite the availability of a product from Genzyme Corp. that will cost substantially less.

On the other side, Sanofi's Genzyme unit maintains Kynamro mipomersen should be attractive to patients because of fewer labeling restrictions on diet, pregnancy and concomitant medications.

And two lipidologists who spoke to BioCentury said they would most often prefer Juxtapid, but could identify scenarios where Kynamro could be the drug of choice for hoFH patients who are not on apheresis and for whom Juxtapid would be contraindicated or ill-advised.

FDAapproved Juxtapid in December, making it the first therapy indicated to treat homozygous familial hypercholesterolemia (hoFH). The drug is an oral small molecule inhibitor of microsomal triglyceride transfer protein (MTP).

The annual price is $235,000 per patient for the 5 and 10 mg doses and $295,000 per patient for the 20, 40 and 60 mg doses.

Kynamro was approved on Jan. 29. It is an injectable second-generation antisense inhibitor of apolipoprotein B-100 (APOB-100) mRNA.

Genzyme, which has exclusive rights from Isis Pharmaceuticals Inc., plans to launch Kynamro this quarter at $3,380 per weekly injection, or about $176,000 per patient annually.

The two products will be competing for roughly 300 hoFH patients in the U.S. These individuals lack functional LDL receptors in the liver, rendering them unable to clear LDL-C from the body.

Patients can have LDL-C levels >600 mg/dL and are at very high risk for coronary artery events and sudden death. Some experience myocardial infarctions before their first birthday, and many have had an MI by age 10. The average life expectancy is 33.

Standard of care includes maximally tolerated doses of lipid-lowering therapies and apheresis when patients have access to the proper facility. Apheresis...

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