Cerus Corporation (Nasdaq:CERS) announced today that the Company has submitted a pre-market approval supplement (PMA-S) to the FDA for a pathogen reduced cryoprecipitated fibrinogen complex with 5-day post thaw storage. This product was previously granted FDA breakthrough device designation based on the potential to improve treatment of massive hemorrhage, a life-threatening medical condition. Trauma is the leading cause of death in the U.S. for people under 45 years of age and approximately 40% of the fatalities are the result of bleeding or its consequence1,2.
“This is a major milestone in our corporate history,” said William ‘Obi’ Greenman, Cerus’ president and chief executive office. “Pathogen-reduced cryoprecipitated fibrinogen complex is manufactured from FDA licensed INTERCEPT plasma. If approved by the FDA, it would be our first therapeutic product candidate. It is being developed specifically to provide a source of fibrinogen and other coagulation factors to treat patients with massive bleeding.”
Severely bleeding patients generally do not receive conventional cryoprecipitate immediately on hospital admission in current massive transfusion protocols or during in-hospital surgical procedures with massive bleeding because it is stored frozen and requires thawing before administration. Furthermore, if unused within four to six hours of thawing, conventional cryoprecipitate must be discarded due to concern for bacterial contamination, and it is wasted3. The high wastage rates due to limited post thaw storage has impeded early use of cryoprecipitate in the treatment of massive hemorrhage, resulting in limited availability at many hospitals.
Cerus’ pathogen-reduced cryoprecipitated fibrinogen complex is designed to be stored for up to five days at room temperature after thaw and to be provided in doses enabling greater ease of use. If approved by the FDA, the room temperature storage would enable hospitals with active trauma centers, major surgical programs, and high-risk obstetric patients to maintain an inventory of thawed pathogen-reduced cryoprecipitated fibrinogen complex. This would allow for immediate availability while minimizing wastage rates due to the longer post-thaw shelf life compared to conventional cryoprecipitate.
“We believe our pathogen-reduced cryoprecipitated fibrinogen complex, if approved, will provide physicians with a critically important option to treat hemorrhaging patients more rapidly when every second counts,” continued Greenman.
“The recent PMA approval for the INTERCEPT Blood System for plasma with alternate plastic disposable kits serves as the basis for our PMA supplement submission,” said Carol Moore, Cerus’ senior vice president of regulatory affairs and quality. “Given the 180-day review process for PMA supplements, we could potentially receive FDA approval for pathogen-reduced cryoprecipitated fibrinogen complex by the end of this calendar year.”
By thawing INTERCEPT treated frozen plasma between 1° and 6° C and separating the precipitated cold-insoluble proteins and the supernatant, two distinct blood components are generated: pathogen reduced cryoprecipitated fibrinogen complex, and pathogen reduced cryoprecipitate-poor plasma. Both products are included in the PMA-S submission.
Pathogen reduced cryoprecipitate-poor plasma has been submitted for approval with an indication to treat thrombotic thrombocytopenic purpura (TTP) and is usually administered by therapeutic plasma exchange (TPE) which is the current practice with conventional cryoprecipitate-poor plasma. TTP is an auto-immune blood disorder which if left untreated has a 90% mortality rate in which a critical enzyme (ADAMTS13) is depleted, resulting in blood clots forming in small blood vessels throughout a patient’s body, thereby blocking blood flow to vital organs4. TPE for TTP is a highly effective therapy, but it requires repeated exposures to large volumes of plasma or cryoprecipitate-poor plasma. Pathogen reduced cryoprecipitate-poor plasma offers the potential to reduce the risk of transfusion-transmitted infections in TTP patients treated with TPE and contains less clot-forming proteins than plasma.
U.S. Commercial Opportunity
In the U.S., trauma is the leading cause of death in individuals under 45 years old and the third leading cause of death overall for all ages1. Approximately 40% of deaths associated with trauma are the result of bleeding or its consequence2. Acute bleeding in other settings includes postpartum hemorrhage and cardiac surgery. Cryoprecipitate is often administered to these patients as an important source of fibrinogen replacement and bleeding control.
According to the 2017 National Blood Collection and Utilization Survey, the number of cryoprecipitate units distributed in the United States between 2015 and 2017 increased by 16.1% to 2.16 million doses or an estimated 700,000+ gram equivalents of fibrinogen.
1. National Vital Statistics Report, Vol. 68, No. 6. https://www.cdc.gov/nchs/data/nvsr/nvsr68/nvsr68_06-508.pdf
2. Curry N, Hopewell S, Doree C, Hyde C, Brohi K, Stanworth S. The acute management of trauma hemorrhage: a systematic review of randomized controlled trials. Crit Care. 2011;15(2):R92.
3. Ramirez-Arcos, S., C. Jenkins, et al. (2017). "Bacteria can proliferate in thawed cryoprecipitate stored at room temperature for longer than 4 h." Vox Sang. 2017; 112(5):477-479.
4. George, J. N. (2010). "How I treat patients with thrombotic thrombocytopenic purpura: 2010." Blood 116: 4060 - 4069.
Cerus Corporation is dedicated solely to safeguarding the world’s blood supply and aims to become the preeminent global blood products company. Based in Concord, California, our employees are dedicated to deploying and supplying vital technologies and pathogen-protected blood components for blood centers, hospitals and ultimately patients who rely on safe blood. With the INTERCEPT Blood System, we are focused on protecting patients by delivering the full complement of reliable products and expertise for transfusion medicine. Cerus develops and markets the INTERCEPT Blood System and remains the only company in the blood transfusion space to earn both CE Mark and FDA approval for pathogen reduction of both platelet and plasma components. Cerus currently markets and sells the INTERCEPT Blood System in the United States, Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. The INTERCEPT Red Blood Cell system is in clinical development. For more information about Cerus, visit www.cerus.com and follow us on LinkedIn.
INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation.
Forward Looking Statements
Except for the historical statements contained herein, this press release contains forward-looking statements related to the recent filing of a PMA-S submission for pathogen reduced-cryoprecipitated fibrinogen complex and pathogen reduced cryoprecipitate-poor plasma, including anticipated regulatory approval, and the timing and the potential claims thereof; and other statements that are not historical facts. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation, risks related to the uncertain and time-consuming development and regulatory process, including that Cerus may be unable to obtain FDA approval in a timely manner or at all; risks with respect to the label claims ultimately approved for pathogen reduced cryoprecipitated fibrinogen complex and pathogen reduced cryoprecipitate-poor plasma; risk that FDA may disagree with Cerus‘ interpretations of the data from its clinical studies, as well as other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including Cerus’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed with the SEC on May 5, 2020. In addition, to the extent that the COVID-19 pandemic adversely affects Cerus’ business and financial results, it may also have the effect of heightening many of the other risks and uncertainties described above. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.