Hyperactivation

Orsini Specialty Pharmacy Selected to be the Exclusive Specialty Pharmacy Partner for VEOPOZ™ (pozelimab-bbfg), the First and Only Treatment for CHAPLE Disease

Retrieved on: 
Wednesday, August 23, 2023
PLE, Regeneron Pharmaceuticals, Orsini, Malnutrition, Edema, Abdominal pain, Hyperactivation, Vomiting, ELK, Dysentery, Patient, CD55, Dentistry

ELK GROVE VILLAGE, Ill., Aug. 23, 2023 /PRNewswire/ -- 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 VEOPOZ™ (pozelimab-bbfg).

Key Points: 
  • ELK GROVE VILLAGE, Ill., Aug. 23, 2023 /PRNewswire/ -- 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 VEOPOZ™ (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 dedicated to providing exceptional care to the patients whose treatment has been entrusted to us."

Pozelimab (C5 Antibody) BLA for Treatment of Children and Adults with Ultra-rare CHAPLE Disease Accepted for FDA Priority Review

Retrieved on: 
Tuesday, February 21, 2023
Rhinitis, Vaccination, CD55, Blood, Edema, Protein, Hospital, Aes, Neisseria meningitidis, Abdominal pain, Rare, C5, Patient, Peripheral edema, Swelling, Biologics license application, Safety, Hives, Human, Hyperactivation, Serum albumin, Thrombus, Vomiting, Food, Risk, Defecation, Malnutrition, Priority, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Regeneron Pharmaceuticals, BLA, Fast Track, Dysentery, Infection, Vaccine, Pharmaceutical industry, Fever

TARRYTOWN, N.Y., Feb. 21, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for pozelimab as a treatment for adults and children as young as 1 year of age with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy). There are currently no approved treatments for CHAPLE, an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. Pozelimab is an investigational fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation. The target action date for the FDA decision is August 20, 2023.

Key Points: 
  • There are currently no approved treatments for CHAPLE, an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system.
  • All patients in the pozelimab trial received vaccination for meningococcal infection prior to treatment with pozelimab.
  • The FDA designated pozelimab for treatment of CHAPLE as a drug for a “rare pediatric disease” in April 2020, with the opportunity for Regeneron to receive a rare pediatric disease priority review voucher should pozelimab be approved for CHAPLE.
  • Pozelimab is currently under clinical development for CHAPLE, and its safety and efficacy have not been evaluated by any regulatory authority.