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FDA Grants Orphan Drug Status to Recombinant FIX Product
 

In November, Biovitrum AB and Syntonix Pharmaceuticals, Inc., announced that they have received orphan-drug designation from the U.S. Food and Drug Administration (FDA) for their novel recombinant factor IX (FIXFc) drug. This FIXFc therapy will be indicated for the control and prevention of hemorrhagic episodes in patients with hemophilia B. The two companies first entered into a co-development and commercialization alliance to develop FIXFc in January 2006.

Syntonix, which is based in Waltham, MA, employs what the company refers to as its “proprietary Fc-fusion technologies” to help create longer-acting biopharmaceutical therapies to treat certain chronic disorders. Biovitrum, with headquarters in Sweden and the United Kingdom, develops and manufactures protein-based therapies for common diseases and those affecting rare disorder populations. Using the technologies of both companies, the objective is to develop a form of FIXFc that exhibits an extended half-life, thereby prolonging the viability of the therapy. An extended half-life may reduce the number of requisite infusions for hemophilia B patients. Pre-clinical studies demonstrated the potential for an extended half-life, while current studies will assess the drug’s safety and pharmacokinetics.
 
“This orphan drug designation is very good news for our existing project; Beyond securing market exclusivity for the drug once it is approved, it will allow for certain benefits, including reduced marketing application and license fees, the ability to work with the Office of Orphan Drugs for protocol assistance, and for appropriate meetings with the Division of Blood Applications,” said Martin Nicklasson, CEO of Biovitrum. “This will help us in our efforts to hopefully bring this improved recombinant Factor IXFc hemophilia compound to those patients who have significant unmet medical needs.”

The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. More than 1,400 drugs and biologics have been designated as orphan drugs. Since 1983 more than 250 of them have been approved for marketing

Source: Biovitrum press release dated November 24, 2008