CSL Behring Receives Orphan-Drug Exclusivity for RiaSTAP(TM), First and Only Approved Treatment of Acute Bleeding Episodes in Patients with Congenital Fibrinogen ...( KING OF PRUSSIA Pa. March 11 /- CSL B...),Health

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, spon
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