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Orphan-Drug Exclusivity Granted for Purified Fibrinogen Concentrate

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.

Source: CSL Behring

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