Dr. Diane Nugent lays foundation for recent FDA approval of Factor XIII to treat potentially fatal bleeding disorder

The U.S. Food and Drug Administration (FDA) recently approved the use of Corifact™, Factor XIII Concentrate (Human), for the routine treatment of congenital factor XIII (FXIII) deficiency. Corifact is the first and only FXIII concentrate approved in the nation.


Congenital FXIII deficiency is a rare and potentially life-threatening inherited bleeding disorder in which blood clots are unstable, due to a lack of the FXIII protein. Consequently, patients are susceptible to recurrent bleeding, including bleeding inside the skull, which can be life threatening. Studies have shown between 25 and 60 percent of FXIII-deficient patients will experience bleeding inside the skull at least once during their lifetime. Additionally, patients can experience bleeding from the umbilical cord after birth, poor wound healing, miscarriages, subcutaneous bleeding and excessive bleeding in joints and muscles following a trauma.


In 1998, Dr. Nugent, CHOC Research Administration and Dr. Nugent’s clinical research team at CHOC Children’s assumed oversight of a compassionate use drug study for FXIII- deficient patients. Since that time, they have supplied treatment to more than 60 patients, and collected safety and efficacy data that helped create the foundation of evidence for the successful FDA approval.


“The CHOC Children’s hematology team is very proud of the role we played in treating patients with this serious disease, while collecting supporting evidence on the efficacy and safety of Corifact. It’s been an amazing opportunity for us to make such a positive impact on the lives of these patients – especially given the rarity of their condition,” says Dr. Nugent.


“Working with 39 performance sites for more than a decade was a logistical challenge, which was overcome by the strength of our relationships with Dr. Nugent and her cadre of committed investigators and institutions from around the country. The Research Institute is proud to have been part of this successful effort to bring this drug to the patients who need it,” states Brent Dethlefs, executive director, CHOC Children’s Research Institute.


Corifact, manufactured by CSL Behring, is an IV infusion administered every 28 days. Clinical studies, including those performed at CHOC, have shown that Corifact immediately increased FXIII levels in the blood. For more information, visit www.corifact.com


CHOC is exclusively committed to the health and well-being of children through clinical expertise, advocacy, outreach and research that brings advanced treatment to pediatric patients. Affiliated with the University of California, Irvine, CHOC’s regional healthcare network includes two state-of-the-art hospitals in Orange and Mission Viejo, several primary and specialty care clinics, a pediatric residency program, and four centers of excellence – The CHOC Children’s Heart, Cancer, Neuroscience, and Orthopaedic Institutes.


Dedicated to the highest performance standards, CHOC earned the Silver Level CAPE Award from the California Council of Excellence, the only children’s hospital in California to ever earn this distinction, and was awarded Magnet designation, the highest nursing honor bestowed to hospitals. Recognized for an extraordinary commitment to high-quality critical care standards, CHOC is the first children’s hospital in the United States to earn the Beacon Award for Excellence.




Media Contact:
Denise Almazan, Director of Public Relations
phone: (714) 509-8680
email: dalmazan@choc.org


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