CONCORD, Calif.--(BUSINESS WIRE)--Cerus Corporation (NASDAQ:CERS) announced today that it has been granted orphan drug status for plasma prepared with the INTERCEPT Blood System for treatment of thrombotic thrombocytopenic purpura (TTP). TTP is a rare, life-threatening blood disorder that typically requires transfusion with large volumes of plasma. Orphan drugs are required to establish safety and efficacy prior to approval by the United States Food and Drug Administration (FDA). The designation facilitates the application process in order to encourage development of new therapies to treat rare conditions and diseases.
“This designation is a significant step toward making INTERCEPT plasma available to a very vulnerable U.S. patient population,” commented Claes Glassell, Cerus' president and chief executive officer. “Patients with TTP are at heightened risk for transfusion transmitted disease given the considerable volumes of plasma used in therapeutic exchange procedures. We look forward to working with the FDA to determine their requirements for a TTP indication approval.”
The incidence of TTP is estimated to be as high of 3500 patients per year in the United States. There are no drugs approved for treatment of TTP, and episodes are associated with significant risk of mortality from widespread blood clotting which restricts blood flow to critical organs such as the brain, kidneys and heart. Over the course of treatment, a patient may receive in excess of 40 liters of plasma, representing exposure to plasma units from over 200 blood donors.
In 2004, Cerus completed a phase 3 study of 35 TTP patients in which almost 3000 units of INTERCEPT plasma were transfused. The results were published in the journal Transfusion in 2006 (Mintz et al. Transfusion 2006;46:1693-1704). The primary endpoint of the trial, comparing the proportion of patients in the test and control groups achieving remission within 30 days after the first therapeutic plasma exchange, was met (82% treated vs. 89% control groups). All secondary efficacy endpoints, comparing time to first remission, relapse rates and plasma exchange volume, were also met. INTERCEPT plasma is currently approved in Europe for use in all indications of plasma for transfusion, including treatment of TTP.
ABOUT CERUS
Cerus Corporation is a biomedical products company focused on commercializing the INTERCEPT Blood System to enhance blood safety. The INTERCEPT system is designed to reduce the risk of transfusion-transmitted diseases by inactivating a broad range of pathogens such as viruses, bacteria and parasites that may be present in donated blood. The nucleic acid targeting mechanism of action enables INTERCEPT treatment to inactivate both established transfusion threats, such as hepatitis B and C, HIV, West Nile virus and bacteria, and is designed to inactivate emerging pathogens such as influenza, malaria and dengue. Cerus currently markets and sells the INTERCEPT Blood System for both platelets and plasma in Europe, the CIS, the Middle East and selected countries in other regions around the world. The INTERCEPT red blood cell system is in clinical development. See http://www.cerus.com for more information.
INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation.
This press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements, including, without limitation, statements relating to Cerus’ expectations regarding the significance of achieving orphan drug status on availability of INTERCEPT-treated plasma. These forward-looking statements are based upon the company’s current expectations. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation, risks associated with conducting clinical trials and obtaining regulatory approval, as well as other risks detailed in the Cerus' filings with, the Securities and Exchange Commission (SEC), including in Cerus' quarterly report on Form 10-Q for the quarter ended Sept 30, 2010, filed with the SEC on November 15, 2010. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Cerus does not undertake any obligation to update any forward-looking statements as a result of new information, future events, changed assumptions or otherwise.