CD55

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."

Veopoz™ (pozelimab-bbfg) Receives FDA Approval as the First Treatment for Children and Adults with CHAPLE Disease

Retrieved on: 
Friday, August 18, 2023
ACIP, Priority review, Vaccination, National Institute of Allergy and Infectious Diseases, Prevalence, Face, Regeneron Pharmaceuticals, Attenuated vaccine, Infection, Advisory Committee on Immunization Practices, Fast Track, Medicine, National Institutes of Health, Bangladesh Technical Education Board, Neisseria meningitidis, Immune system, Meningococcal disease, Patient, Risk, CD55, Blood, NIAID, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Health insurance coverage in the United States, BLA, NIH, Food, C5, Disease, Pharmaceutical industry, Vaccine

TARRYTOWN, N.Y., Aug. 18, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has approved Veopoz™ (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed. FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.

Key Points: 
  • With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed.
  • FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.
  • Veopoz was reviewed under Priority Review, and the Company received a Rare Pediatric Disease Priority Review voucher upon approval.
  • Veopoz was previously granted Rare Pediatric Disease designation, Orphan Disease designation and Fast Track designation.

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.

Paroxysmal Nocturnal Hemoglobinuria Market to Climb Moderately at a CAGR of 7.6% During the Study Period (2019-2032), Assesses DelveInsight

Retrieved on: 
Monday, January 23, 2023
Safety, Therapy, CD55, EU4, Nitric oxide, LAS, Hemolysis, United Kingdom, Arrowhead Pharmaceuticals, III, CD59, Diagnosis, Death, BioCryst Pharmaceuticals, Immunosuppression, Eculizumab, Urine, Novartis, Haematopoietic system, Roche, Flow cytometry, GPI, Hemoglobinuria, HSC, US, Bone marrow failure, Drug development, FDA, Paroxysmal nocturnal hemoglobinuria, PIGA, Alnylam Pharmaceuticals, Venous thrombosis, SCR, Regeneron Pharmaceuticals, Patient, Alexion Pharmaceuticals, Czech Republic, Hemosiderin, Disease, PNH, Thrombosis, Pharmaceutical industry, Lithium, Medical device, MorphoSys

LAS VEGAS, Jan. 23, 2023 /PRNewswire/ -- DelveInsight's Paroxysmal Nocturnal Hemoglobinuria Market Insights report includes a comprehensive understanding of current treatment practices, paroxysmal nocturnal hemoglobinuria emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Points: 
  • In December 2022, Novartis released the efficacy and adverse event data from the phase III APPLY-PNH trial in paroxysmal nocturnal hemoglobinuria.
  • In September 2022, efficacy and safety data from a phase III trial in paroxysmal nocturnal hemoglobinuria were released by Alexion.
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological disorder characterized by intravascular hemolysis, hemoglobinuria, hemolysis, and venous thrombosis.
  • To know more about paroxysmal nocturnal hemoglobinuria treatment guidelines, visit @ Paroxysmal Nocturnal Hemoglobinuria Management

Pan Cancer T Appoints Dr. Rachel Abbott as Chief Scientific Officer

Retrieved on: 
Tuesday, January 17, 2023
Dark, University, Pan-Cancer Analysis, Erasmus, Immunotherapy, Aeris Naviter AN-2 Enara, Erasmus MC, CD55, Head, Ligand, Therapy, Patient, Laboratory, TCR, European, Doctor of Philosophy, Vaccine

ROTTERDAM, January 17, 2023 - Pan Cancer T B.V. , a biotech spin-off from Erasmus MC dedicated to the discovery and development of next-generation TCR-T therapies against solid tumors, today announced the appointment of Dr. Rachel Abbott as Chief Scientific Officer.

Key Points: 
  • ROTTERDAM, January 17, 2023 - Pan Cancer T B.V. , a biotech spin-off from Erasmus MC dedicated to the discovery and development of next-generation TCR-T therapies against solid tumors, today announced the appointment of Dr. Rachel Abbott as Chief Scientific Officer.
  • Prior to joining Pan Cancer T, Rachel Abbott had been Senior Director, Head of TCR Pipeline and Dark Antigen Research at Enara Bio from March 2021 to January 2023.
  • "We warmly welcome Rachel Abbott to our management team,” said Katrien Reynders-Frederix, CEO of Pan Cancer T. "Rachel has tremendous industry experience, including developing T cell therapies, leading scientific research teams and managing collaborations with big pharma and academic institutions.
  • "I am honored to take over the position as Pan Cancer T´s Chief Scientific Officer,” said Dr. Rachel Abbott.