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.
Source: Cerus Corporation