Healthcare Industry News:  hemophilia A 

Biopharmaceuticals FDA

 News Release - November 24, 2008

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

STOCKHOLM, Sweden--(Healthcare Sales & Marketing Network)--Biovitrum AB (publ) (STO: BVT) and Syntonix Pharmaceuticals, Inc., a subsidiary of Biogen Idec (NasdaqGS:BIIB ) today 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. The current global market for Factor IX products is estimated at $600 MM USD annually. According to a recent analysis, the market is expected to grow to $1.5 BN USD annually by 2015.

"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.

"We are pleased to have achieved this important goal for the FIXFc program, which we are developing in a 50/50 partnership with Biovitrum. The purpose of both the FIXFc and Factor VIIIFc programs is to improve the lives of hemophilia patients and their families," said Matt Ottmer, Vice President of Syntonix.

About Hemophilia

Hemophilia is a rare hereditary disorder in which the ability of patients' blood to clot is impaired. As a result, the patient suffers from excessive bleeding and uncontrolled internal bleeding, leading to pain and eventual permanent damage to joints and muscles. One form, Hemophilia B results from mutations that impair the production of Factor IX. It has been reported that even with "proper treatment" the life expectancy of hemophilia patients is about 10 years less than for individuals without hemophilia. Increasingly, the normal mode of treatment for younger patients is a prophylaxis regimen where patients are infused two or three times per week to maintain a better circulating level of coagulation factor. Long term studies demonstrate that such regimens greatly reduce if not eliminate progressive joint deterioration.

About SynFusionTM Technology

The SynFusion technology is based on Syntonix's proprietary Fc-fusion technologies to create longer-acting biopharmaceuticals. Well-known and validated traditional Fc-fusion drugs, such as EnbrelĀ® (etanercept) for the treatment of rheumatoid arthritis, consist of two copies of a biopharmaceutical linked to the Fc region of an antibody to improve pharmacokinetics, solubility, and production efficiency. SynFusion drugs consist of a novel Fc-fusion construct, called a monomer that links only a single copy of the drug to the Fc region on an antibody to optimize the pharmacokinetic and pharmacodynamic properties of the biopharmaceutical when compared to traditional Fc-fusion constructs.

About Biovitrum

Biovitrum is a pharmaceutical company with operations in Sweden and in the UK. The company markets a range of specialist pharmaceuticals primarily in the Nordic countries. Using its expertise and experience Biovitrum takes scientific innovation all the way to the market and to specialist indication patients with significant medical need. Research expertise and capabilities include development and production of biotechnology therapeutics, as well as small molecule discovery and development. With revenues of approximately SEK 1.3 billion and around 500 employees, Biovitrum is a significant European specialty pharmaceutical player. Biovitrum's share is listed on the OMX Nordic Exchange in Stockholm. For more information go to

About Syntonix

Syntonix Pharmaceuticals, Inc. (Waltham, MA, USA) is a wholly-owned subsidiary of Biogen Idec. Syntonix is developing next generation biopharmaceuticals that enable better treatment options for patients with devastating chronic diseases such as hemophilia And autoimmune disorders. The company applies its core technologies to develop long-acting biopharmaceuticals that may be injected less frequently, and to discover novel drugs to treat antibody-mediated autoimmune and inflammatory disorders. The resulting proteins, peptides and antibodies are being commercialized through internal development programs and collaborations with biotechnology and pharmaceutical partners. More information is available at

Safe Harbor/Forward-looking Statements

This press release contains forward-looking statements regarding the market for Factor IX products and the impact of orphan drug designation. These statements are based on current beliefs and expectations. The development and commercial potential of the FIXFc compound is subject to a number of risks and uncertainties, including the potential safety and efficacy profile of the FIXFc compound and the projected timeline and cost for completing clinical trials of the FIXFc compound. Drug development and commercialization involves a high degree of risk.

Biovitrum AB (publ) may be required to disclose the information provided herein pursuant to the Swedish Securities Markets Act. The information was provided for public release on November 24, 2008 at 02:00 p.m. CET.

Source: Biovitrum

Issuer of this News Release is solely responsible for its content.
Please address inquiries directly to the issuing company.

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