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Orphan Drugs: FDA Approval For Hemophilia A

June 7, 2014

On June 6th, the FDA approves Biogen Idec’s orphan drug Eloctate, Antihemophilic Factor (Recombinant), Fc fusion protein, for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in both adults and children with Hemophilia A. Eloctate is the first Hemophilia A treatment that results in less frequent injections when used for the reduction or prevention of bleeding, because of its prolonged circulation in the body.

FDA Orphan Drug Designation (ODD) Database Record

Generic Name: recombinant human coagulation factor VIII Fc fusion protein
Trade Name: n/a
Date Designated: 11-23-2010
Orphan Designation: Treatment of hemophilia A
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Biogen Idec Hemophilia, Inc. 9 Fourth Avenue Waltham, MA 02451 The sponsor address listed is the last reported by the sponsor to OOPD.

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According to Biogen Idec’s Press Release, Hemophilia A:

•   Occurs in approximately 1 in 5,000 male births

•   Effects approximately 16,000 patients in the US

•   Effects approximately 142,000 patients worldwide.

Biogen Idec’s orphan drug Alprolix is approved in March 2014 in the US and Canada for use in both children and adults for Hemophilia B. It is also the first approved long-acting recombinant coagulation Factor IX concentrate for Hemophilia B. Alprolix is made up of the Factor IX molecule linked to a protein fragment Fc (found in antibodies), which makes the product last longer in circulation. This results in less frequent injections for patients. Alprolix is currently under review by regulatory authorities in Australia and Japan.

Biogen Idec and Swedish Biovitrum (Sobi) are collaborating in both the development and commercialization of Eloctate and Alprolix.

Please Note: FDA Official Logo from FDA website.

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4 Comments
  1. Its fascinating, and a serious breakthrough. Giving factor daily can be exhausting. I’m excited for this!

    • Hi, Thanks for visiting the Blog and adding your voice. Yes, it is great that with this approval, giving factor daily may be eliminated. Thanks, Ann

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