Navigation Links
CSL Behring Receives FDA Orphan Drug Designation for rVIIa-FP, a Novel Therapy in Development to Treat Hemophilia A and Hemophilia B Patients Who Have Inhibitors
Date:2/16/2012

KING OF PRUSSIA, Pa., Feb. 16, 2012 /PRNewswire/ -- CSL Behring announced today that the company has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its novel recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). The Orphan Drug Designation is granted for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia and inhibitors to coagulation factor VIII or IX. CSL Behring is developing this therapy in collaboration with its parent company CSL Limited (ASX: CSL).

The CSL Behring rVIIa-FP clinical program will soon be initiated and intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients who have hemophilia A and B with inhibitors.

"CSL Behring's albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins because of its high tolerability, inherently long half-life, low potential for immunogenic reactions and known mechanism of clearance," said Russell Basser, M.D., Senior Vice President, Global Clinical R&D at CSL Behring. "CSL Behring's rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis. We welcome Orphan Drug Designation for our rVIIa-FP and will work closely with the FDA to make this important therapy available for people in the U.S. with hemophilia A and hemophilia B with inhibitors."

The FDA's Orphan Drug Designation program provides orphan status to unique drugs and biologics, defined as those intended for the safe and effective treatment or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the product for important tax credits, elimination of FDA license application fees and certain marketing incentives.

CSL Behring's rVIIa-FP was granted Orphan Drug Designations (ODD) by the European Commission in May, 2011.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective.  Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which renders replacement therapy ineffective.  This can occur in up to 25 percent of hemophilia A patients and around 5 percent of hemophilia B patients.  One treatment option for these patients is recombinant activated factor VII (called a bypassing agent) which can be used to achieve hemostasis without the need for factor VIII or IX. 

About the recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP)
Preclinical studies have confirmed that CSL Behring's rVIIa-FP has favorable pharmacokinetic properties compared with the existing recombinant FVIIa product. Significant increases in half-life have been observed across all animal species. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with currently available product.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX:CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.


'/>"/>
SOURCE CSL Behring
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. CSL Behring Receives FDA Approval of Expanded Label on Berinert® for Self-administration and Treatment of Acute Laryngeal Attacks of Hereditary Angioedema
2. CSL Behring Seeks Proposals for Interlaken Leadership Awards, Supporting Innovative Research Into Neuroimmunology
3. CSL Behring Receives EU Orphan Drug Designations for rVIIa-FP for Hemophilia A and B Treatment
4. National Organization for Rare Disorders Presents CSL Behring With 2011 Corporate Award
5. CSL Behring Marks World Hemophilia Day with Coagulation Factor Donation to World Federation of Hemophilia
6. CSL Behring Medical Product Donation Helps Meet Critical Need for Ongoing Cholera Relief Effort in Haiti
7. CSL Behring Announces Completion of National Marketing Authorizations of Berinert® After MRP in 23 European Countries
8. CSL Behring Announces FDA Approval of Berinert(R), First and Only Therapy Approved for the Treatment of Acute Abdominal and Facial Attacks of Hereditary Angioedema in U.S.
9. CSL Behring Receives Orphan-Drug Exclusivity for RiaSTAP(TM), First and Only Approved Treatment of Acute Bleeding Episodes in Patients with Congenital Fibrinogen Deficiency
10. Over $10 Million of CSL Behrings Privigen Was Sold in the U.S. During the Third Quarter of 2008
11. CSL Behring Submits BLA Requesting Approval of C1-Esterase Inhibitor for the Treatment of Hereditary Angioedema
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/5/2017)... Oramed Pharmaceuticals Inc. (NASDAQ: ORMP ) (TASE: ... on the development of oral drug delivery systems, announced ... the U.S. Food and Drug Administration (FDA) regarding ORMD-0801, ... At the meeting, the FDA gave ... ORMD-0801, would be a Biologics License Application (BLA).  Such ...
(Date:9/1/2017)... , Sept. 1, 2017  Bayer will present the latest ... Society for Medical Oncology (ESMO) 2017 Congress, September 8-12 in ... include new preclinical and clinical data on Bayer,s marketed portfolio ... pipeline projects. "We ... minds in cancer research at ESMO," said Carsten Brunn ...
(Date:8/29/2017)... , Aug. 29, 2017 In a ... security, and regulatory compliance for veterinary practices of all ... a new partnership that makes TITAN,s expertise in physical ... available to Cubex,s clients nationally. ... buying and handling controlled substances is at risk today," ...
Breaking Medicine Technology:
(Date:9/19/2017)... ... September 19, 2017 , ... ... a neurosurgeon, has received a big bump in sales after recently going ... currently available on the Wal-Mart Stores-owned Jet.com, StackedNutrtion.com, RonnieColemanNutrition.com, RevNutrition.com and Go4ItNutrition.com. ...
(Date:9/19/2017)... Glen Cove, New York (PRWEB) , ... September 19, 2017 , ... ... that its dentist, Dr. Andrew Sami, was recently named one of the best dentists ... the leading lifestyle magazine for dental professionals nationwide. Every fall, the magazine features the ...
(Date:9/19/2017)... (PRWEB) , ... September 19, 2017 , ... ... update their mobile app offering. The new app provides a modern design with ... the overall shopping experience. , The fred’s Pharmacy mobile app includes hundreds of ...
(Date:9/18/2017)... ... September 19, 2017 , ... Food ... market , expected to grow at the fastest CAGR of 9.1% over the ... Food Protein Trends & Growth Opportunities in Asia’ – highlighting alternative proteins, sustainable ...
(Date:9/18/2017)... ... September 18, 2017 , ... Allegheny Health Network’s (AHN) string of standout ... fifth consecutive year, AHN earned more honors than any health care provider in the ... dinner Sept. 14 at the Omni William Penn Hotel in downtown Pittsburgh included:, ...
Breaking Medicine News(10 mins):