Biovitrum's and Syntonix's Novel Factor IXFc for Hemophilia B has Received Orphan Drug Designation From the FDA

27-Nov-2008 - Switzerland

Biovitrum AB and Syntonix Pharmaceuticals, Inc., a subsidiary of Biogen Idec announced that the company's FIXFc compound for the control and prevention of hemorrhagic episodes in patients with hemophilia B has received an orphan-drug designation from the US food and Drug Administration (FDA). The FIXFc compound is a recombinant protein in early-stage development for the treatment of hemophilia B, a hereditary bleeding disorder characterized by impaired production of factor IX and the blood's inability to coagulate. The Fc fusion with factor IX is intended to prolong the effect of the compound, the goal being that patients will need less frequent prophylactic treatments than currently existing therapies, which require two to four administrations per week.

The companies' FIXFc has previously received an EMEA (COMP) orphan medicinal product designation for the treatment of hemophilia B. Pre-clinical studies have shown that this FIXFc compound may have an extended half-life, and a study is currently ongoing to determine the compound's safety and pharmacokinetics in hemophilia B patients.

"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. This will help us in our efforts to hopefully bring this improved recombinant Factor IXFc hemophilia compound to those patients who have significant unment medical needs." said Martin Nicklasson, CEO of Biovitrum.

https://www.bionity.com/en/news/90346/biovitrum-s-and-syntonix-s-novel-factor-ixfc-for-hemophilia-b-has-received-orphan-drug-designation-from-the-fda.html