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Why Is Aegerion Pharmaceuticals’ Orphan Drug Juxtapid (Lomitapide) In The News (December 24, 2012) ?

December 24, 2012

 On December 24, 2012, Aegerion Pharmaceuticals announces that the FDA approves orphan drug Juxtapid (Lomitapide) capsules. Juxtapid’s approval comes a few days earlier than the scheduled FDA  PDUFA date of December 28 – 29, 2012. Juxtapid is  Aegerion Pharmaceuticals 1st FDA approved product. Aegerion Pharmaceuticals plans to start selling Juxtapid in January 2013.

Juxtapid, a once-daily pill, is approved as an “adjunct to a low-fat diet and other lipid-lowering treatments, including apheresis where available , to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and high-density-lipoprotein cholesterol (non-HDL) in patients with” Homozygous Familial Hypercholesterolemia (HoFH).

Juxtapid contains a Black Box Warning, the FDA’s most severe safety warning, identifying the risk of hepatic toxicity. The approval is based on the drug’s pivotal Phase III study which evaluated safety and effectiveness of the medicine. Aegerion Pharmaceuticals is required to conduct a long-term postmrketing observational study to monitor Juxtapid’s effects on liver enzymes and hepatotoxicity. At the October 2012 meeting of the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee, the company agreed to this study.

Because of the liver toxicity risk, Juxtapid is available only through a restricted program, Juxtapid Risk Evaluation and Mitigation Strategy (REMS) Program. Aegerion Pharmaceuticals will certify all health care providers and the pharmacies that will dispense Juxtapid.  The REMS also now requires patients to have a “clinical or laboratory diagnosis consistent with HoFH,” but does not specify how the diagnosis is to be made. Previously, the REMS did not require a specific diagnosis to dispense the drug. As part of the Juxtapid launch, the company provides a “comprehensive support services program” for patients and providers.

Per a December 24, 2012 Forbes.com article titled, “Aegerion Cholesterol Drug Approved. Will it treat 300 Patients OR 3000 ? ” Juxtapid  :

  1. Will Cost $200,000 – $300,000/year/patient
  2. Will Be sold only through a single mail order pharmacy
  3. Estimates of 300 – 3,000 patients in the US for HoFH market
  4. Size of the HoFH market is key to both Juxtapid and Isis Pharmaceuticals’ HoFH drug Kynamro (scheduled PDUFA date of January 29, 2013)
  5. Size of the HoFH market may be more important to both drugs than which one has the better efficacy and safety profile.

Aegerion Pharmaceuticals is to present at the J.P. Morgan Healthcare Conference beginning January 7, 2013, in San Francesco. The company will provide additional information on pricing at this conference.

References

Bloomberg BusinessWeek.com December 24, 2012 article titled, “Aegerion Drug Wins Approval for Rare Cholesterol Illness”

The Familial Hypercholesterolemia (FH) Foundation.

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

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