CSL Behring Receives Orphan-Drug Exclusivity for RiaSTAP(TM)
Wed, 03/11/2009 - 9:12am
CSL Behring has beeninformed by the FDA that becauseRiaSTAP(TM) is the first FDA-approved treatment of acute bleeding episodes inpatients with congenital fibrinogen deficiency, the company is entitled toseven years of orphan-drug exclusivity. RiaSTAP was approved by the FDA inJanuary and is indicated for the treatment of acute bleeding episodes inpatients with congenital fibrinogen deficiency, including afibrinogenemia andhypofibrinogenemia. Approval was based on a pivotal phase II prospective,open-label pharmacokinetic (PK) and safety study using maximum clot firmnessas a surrogate endpoint for hemostatic efficacy. "As a leader in developing safe, effective and high-quality biologictherapies, CSL Behring is committed to meeting the needs of patients with raredisorders," said Paul Perreault, Executive Vice President of CSL Behring'sWorldwide Commercial Operations. "As the only company with an FDA approvedtreatment for congenital fibrinogen deficiency, CSL Behring will work closelywith the healthcare community to ensure that all patients have access toRiaSTAP." In its letter to CSL Behring, the FDA expressed the agency's gratitudethat the company had developed RiaSTAP, explaining: "The entire premise of theorphan products program is based on the realization that the resources andcommitment devoted to the development of drugs for 'orphan' populations maynot provide financial return to their sponsors." Congenital fibrinogen deficiency is a rare, potentially life-threateningbleeding disorder that affects an estimated one person per million, with anestimated prevalence in the U.S. of approximately 300 patients. Symptoms ofcongenital fibrinogen deficiency include excessive bleeding following injury,bruising, bleeding of the umbilical cord at birth and from the site of theumbilical stump in a newborn, spontaneous bleeding and bone, joint or tissuehemorrhage. Diminished concentrations of fibrinogen, also called Factor I(one), limit the body's ability to form a clot. Certain fibrinogen levelsusually indicate normal blood clotting ability. To determine fibrinogen levelsand confirm a diagnosis, blood coagulation testing is needed.RiaSTAP is a purified fibrinogen concentrate that undergoes virusinactivation and removal for safety assurance. There have been more than 1million units sold worldwide (marketed outside the U.S. under the trade nameHaemocomplettan(R) P). CSL Behring is studying RiaSTAP in an ongoingpost-marketing commitment study to further demonstrate safety and hemostaticefficacy.