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Good News For Genzyme And Isis Pharmaceuticals’ Orphan Drug Kynamro

January 29, 2013

For the past few months, the Orphan Druganaut Blog has followed the journey of Genzyme and Isis Pharmaceuticals’ orphan drug Kynamro (Mipomersen) for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), from the FDA Advisory Committee recommendation, to the EMA CHMP negative recommendation, and finally to the FDA approval today. HoFH affects approximately 1/1,000,000 people in the U.S.

Kynamro carries a Boxed Warning on the serious risk of liver toxicity because of “its association with liver enzyme abnormalities and accumulation of fat in the liver, which could lead to progressive liver disease with chronic use”. Kynamro is approved with a Risk Evaluation and Mitigation Strategy (REMS) with elements to ensure safe use – pharmacy and provider certification, and documentation of safe-use conditions (prescription authorization form for each new prescription). The FDA is requiring four postmarketing studies for Kynamro.

Genzyme said it will identify pricing information when Kynamro is launched, but no launch timeline has been given yet. The approval results in the $25 million milestone payment from Genzyme to Isis Pharmaceuticals. Genzyme has the rights to Kynamro.

Isis Pharmaceuticals will have a conference call and webcast to provide an update on the FDA approval of Kynamro on Wednesday, January 30, 2013, at 9:30 AM ET.

Kynamro and Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide) are in a battle for the same indication in the United States, HoFH. Both orphan drugs have been nearly parallel in development in the pipeline. Juxtapid was approved in December 2012 for HoFH and costs $235,000 – $295,000/patient/year. Both drugs are the first to be developed and approved for HoFH. Juxtapid is a once-a-day pill, while Kynamro is injected once a week.

It will be interesting to see how the drug companies approach their marketing and launch strategies for the same indication in the U.S.


FDA January 29, 2013, Press Release titled, “FDA approves new orphan drug Knyamro to treat inherited cholesterol disorder” January 29, 2013 article titled, “FDA Approves Mipomersen for Homozygous Familial Hypercholeterolemia”

New York Times January 29, 2013 article titled, “FDA Approves Genetic Drug to Treat Rare Disease”

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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