Genzyme and Isis get FDA approval for drug that treats rare cholesterol disease

Genzyme, now a Cambridge-based division of French drug maker Sanofi SA, and partner Isis Pharmaceuticals Inc. said Wednesday that they have gotten regulatory approval to market a treatment for a rare cholesterol disease that afflicts about one in every 1 million people.

Specifically, Genzyme and Isis said that the US Food and Drug Administration has approved its application for an injectable drug called Kynamro

David Meeker. File photo. (Aram Boghosian for The Boston Globe)

. The drug will be used to treat patients with homozygous familial hypercholesterolemia, or HoFH, a rare inherited condition that makes the body unable to remove LDL cholesterol. For those with HoFH, heart attacks and death often occur before age 30, Genzyme and California-based Isis said in their press release.


The FDA approval triggers a $25 million milestone payment to Isis from Genzyme, the release added.

“Today’s FDA approval of Kynamro is great news for patients with HoFH who are in need of additional treatment options for this rare, and often under-diagnosed disease,’’ Genzyme president and chief executive David Meeker, M.D., said in a statement. “As the leader in treatments for rare diseases, we are pleased to bring our expertise to HoFH patients living with this serious condition to better help them manage their disease.’’

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