ELK GROVE VILLAGE, IL – Aug. 23, 2023 – Orsini Specialty Pharmacy (“Orsini”), a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced today that Regeneron Pharmaceuticals, Inc. (“Regeneron”) has chosen Orsini to be the exclusive specialty pharmacy partner for VEOPOZTM (pozelimab-bbfg). A monoclonal antibody, VEOPOZ is the first and only treatment for those living with CHAPLE disease, an ultra-rare and life-threatening hereditary disease caused by an overactivation of the complement system. VEOPOZ is approved for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.
Orsini is now the exclusive specialty pharmacy for two of Regeneron’s ultra-rare disease treatments, the other being EVKEEZA® (evinacumab-dgnb) for the treatment of adult and pediatric patients, aged 5 years and older, with homozygous familial hypercholesterolemia (HoFH).
CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy) is characterized by an inability to regulate complement activity because of mutations in the CD55 gene. In most patients, this leads to potentially life-threatening symptoms beginning in infancy, including abdominal pain, bloody diarrhea, vomiting, malnutrition, slow growth, and edema. There are fewer than 100 patients worldwide who are known to have CHAPLE disease.
“Orsini is excited to partner with Regeneron in bringing hope to those living with CHAPLE, who had no treatment options before now, and honored to serve as the exclusive specialty pharmacy of another therapy for an ultra-rare disease,” Brandon Tom, Orsini’s President and Chief Executive Officer, said. “Orsini is dedicated to providing exceptional care to the patients whose treatment has been entrusted to us.”
Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A. Individuals with the most severe form of hemophilia A makeup approximately 50% of the hemophilia A population.
About Orsini Specialty Pharmacy
Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, health care providers and payers to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini delivers customized solutions that simplify how patients connect to advanced therapies. Orsini’s high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.
Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC, and the National Association of Boards of Pharmacy (NABP). Orsini has earned URAC’s Rare Disease Pharmacy Center of Excellence Designation and ACHC’s Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373, email us at media@orsinihc.com or visit www.orsini.com/.
For more information about VEOPOZ, see the VEOPOZ Full Prescribing Information.