Navigation Links
CSL Behring Receives Orphan-Drug Exclusivity for RiaSTAP(TM), First and Only Approved Treatment of Acute Bleeding Episodes in Patients with Congenital Fibrinogen Deficiency

KING OF PRUSSIA, Pa., March 11 /PRNewswire/ -- CSL Behring has been informed by the U.S. Food and Drug Administration (FDA) that because RiaSTAP(TM) is the first FDA-approved treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency, the company is entitled to seven years of orphan-drug exclusivity. RiaSTAP was approved by the FDA in January and is indicated for the treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. Approval was based on a pivotal phase II prospective, open-label pharmacokinetic (PK) and safety study using maximum clot firmness as a surrogate endpoint for hemostatic efficacy.

"As a leader in developing safe, effective and high-quality biologic therapies, CSL Behring is committed to meeting the needs of patients with rare disorders," said Paul Perreault, Executive Vice President of CSL Behring's Worldwide Commercial Operations. "As the only company with an FDA approved treatment for congenital fibrinogen deficiency, CSL Behring will work closely with the healthcare community to ensure that all patients have access to RiaSTAP."

In its letter to CSL Behring, the FDA expressed the agency's gratitude that the company had developed RiaSTAP, explaining: "The entire premise of the orphan products program is based on the realization that the resources and commitment devoted to the development of drugs for 'orphan' populations may not provide financial return to their sponsors."

Congenital fibrinogen deficiency is a rare, potentially life-threatening bleeding disorder that affects an estimated one person per million, with an estimated prevalence in the U.S. of approximately 300 patients. Symptoms of congenital fibrinogen deficiency include excessive bleeding following injury, bruising, bleeding of the umbilical cord at birth and from the site of the umbilical stump in a newborn, spontaneous bleeding and bone, joint or tissue hemorrhage. Diminished concentrations of fibrinogen, also called Factor I (one), limit the body's ability to form a clot. Certain fibrinogen levels usually indicate normal blood clotting ability. To determine fibrinogen levels and confirm a diagnosis, blood coagulation testing is needed.

RiaSTAP is a purified fibrinogen concentrate that undergoes virus inactivation and removal for safety assurance. There have been more than 1 million units sold worldwide (marketed outside the U.S. under the trade name Haemocomplettan(R) P). CSL Behring is studying RiaSTAP in an ongoing post-marketing commitment study to further demonstrate safety and hemostatic efficacy.

About RiaSTAP

RiaSTAP is a purified fibrinogen concentrate indicated for the treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. RiaSTAP is not indicated for dysfibrinogenemia. RiaSTAP is contraindicated in individuals who have had severe immediate hypersensitivity reactions, including anaphylaxis to RiaSTAP or its components.

Physicians should monitor patients for early signs of allergic or hypersensitivity reactions and if necessary, discontinue administration and institute appropriate treatment. Thrombotic events have been reported in patients receiving RiaSTAP. Physicians should weigh the benefits of administration versus the risks of thrombosis.

RiaSTAP is made from pooled human plasma. Products made from human plasma may contain infectious agents (e.g., viruses and theoretically the Creutzfeldt-Jakob disease agent (CJD) that can cause disease. The risk that such products will transmit an infectious agent has been reduced by screening plasma donors for prior exposure to certain viruses, by testing for the presence of certain current virus infections, and by a process demonstrated to inactivate and/or remove certain viruses during manufacturing. Despite these measures, such products may still potentially transmit disease. There is also the possibility that unknown infectious agents may be present in such products.

The most serious adverse reactions that have been reported in clinical studies or through postmarketing surveillance following RiaSTAP treatment are allergic-anaphylactic reactions and thromboembolic episodes, including myocardial infarction, pulmonary embolism, deep vein thrombosis and arterial thrombosis. The most common adverse reactions that have been reported in clinical studies or through postmarketing surveillance following RiaSTAP treatment are allergic reactions and generalized reactions such as chills, fever, nausea and vomiting.

For more information, visit

About CSL Behring

CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients' lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company's therapies are used in the treatment of immune deficiency disorders, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, ZLB Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit

    Sheila A. Burke, Director, Communications & Public Relations
    Worldwide Commercial Operations
    CSL Behring
    610-878-4209 (o)
    484-919-2618 (c)

    Brian Thompson
    908-234-9900 (o)

Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Over $10 Million of CSL Behrings Privigen Was Sold in the U.S. During the Third Quarter of 2008
2. CSL Behring Submits BLA Requesting Approval of C1-Esterase Inhibitor for the Treatment of Hereditary Angioedema
3. CSL Behring Study in Animal Models Shows Feasibility of Developing a Half-Life Extended Recombinant FVIIa that Retains Biologic Activity
4. CSL Behring Reaches Primary Endpoint in Clinical Study of C1-INH for Treatment of Hereditary Angioedema
5. Childrens Hospital Receives $6.7 Million Grant From Gates Foundation to Study Influenza Vaccine in Pregnant Women in Asia
6. deCODE Receives California Clinical Laboratory License
7. Omeros Receives $3.1 Million Milestone Payment from The Stanley Medical Research Institute for Schizophrenia Program
8. Phico Therapeutics Receives GBP1 Million Award From the Wellcome Trust to Take Anti-MRSA Nasal Product Through Early Clinical Trials
9. PEAK Surgical Receives 510(k) Clearance to Market PEAK(R) Surgery System for Use in Expanded Indications
10. Kiadis Pharma Receives Two Orphan Drug Designations for Reviroc(TM) From the FDA
11. Vical Receives Milestone Payment from Merck & Co., Inc. Triggered by Planned Initiation of Investigational Cancer DNA Vaccine Trial
Post Your Comments:
(Date:11/25/2015)... 2015 Kitov Pharma ceuticals ... a biopharmaceutical company focused on the development of therapeutic ... today announced the closing of its previously announced underwritten ... ), each representing 20 ordinary shares of the Company, ... ADSs and warrants were issued in a fixed combination ...
(Date:11/25/2015)... Endo International plc (NASDAQ: ENDP ) (TSX: ... and CEO, will discuss corporate updates at the 27 th ... York on Wednesday, December 2, 2015 at 1 ... Relations, and then the link to the event. Participants should ... to visit the site and download any streaming media software ...
(Date:11/25/2015)... -- Developmental, commercial, and regulatory/legal strategies ... pharmaceutical products, says GBI Research . ... all play a key role in boosting the profitability ... . --> Developmental, commercial, and ... the profitability of pharmaceutical products, says GBI Research ...
Breaking Medicine Technology:
(Date:11/25/2015)... ... 25, 2015 , ... In an ongoing Clinical Study conducted by an independent ... UV Angel is evaluating the efficacy of its product and its disinfection protocol. This ... beds) from May 2014 through October 2015 at a 360-bed, acute-care, academic medical center ...
(Date:11/25/2015)... ... November 25, 2015 , ... In honor of Pulmonary ... individuals and groups responsible for advancing care for pulmonary hypertension (PH) patients and ... the public, will receive special recognition throughout 2016 as part of PHA’s 25th ...
(Date:11/25/2015)... ... ... Many people know of the common symptoms of low thyroid hormone (also known ... people who find their cholesterol levels and weight are creeping up are more likely ... don’t have any of the other symptoms. , Thyroid hormone plays a major role ...
(Date:11/25/2015)... ... November 25, 2015 , ... Bunion ... in an early celebration of the early holiday shopping season. Starting Wednesday November ... (normally $33.95 ea). Black Friday promotional pricing is in addition to any automatic ...
(Date:11/24/2015)... ... November 24, 2015 , ... New patients who wish to seek ... dental implants at her Mississauga, ON practice. Dr. Williams has been providing ... of dental implants. , Missing teeth can lead to a variety of complications ...
Breaking Medicine News(10 mins):