FDA Approves Pozelimab (Veopoz) as First Treatment for CHAPLE Disease

The US Food and Drug Administration (FDA) has approved pozelimab-bbfg (Veopoz) for the treatment of for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease.

Announced by Regeneron on August 18, 2023, the approval makes pozelimab-bbfg the first and only treatment indicated specifically for CHAPLE disease, which is also known as CD55-deficient protein-losing enteropathy and can cause potentially life-threatening gastrointestinal and cardiovascular symptoms.¹

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, MD, chief of Molecular Development of the Immune System Section and codirector of the Clinical Genomics Program at the National Institute of Allergy and Infectious Disease in the National Institutes of Health.¹ “As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that pozelimab achieves in those suffering from CHAPLE. The approval of pozelimab is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”

A disease characterized by the inability to regulate complement activity due to mutations in their CD55 gene, the release from Regeneron suggests there are fewer than 10 patients with CHAPLE disease identified in the US. A fully human monoclonal antibody designed to target complement factor C5, the approval of pozelimab-bbfg marks the 10th FDA-approved medicine invented by Regeneron and is based on the results of phase 2/3 open-label trial conducted in 10 patients aged 3-19 years of age, with a median age of 8.5 years.¹ Launched in 2020, protocol for the trial required all patients to receive a single loading dose of pozelimab-bbfg 30 mg/kg intravenously on day 1 followed by subcutaneous weekly weight-based doses of pozelimab.^1,2

Results of the trial suggested all 10 patients achieved normalization of serum albumin and serum IgG concentrations by week 12 and maintained these concentrations through at least 72 weeks of treatment. A total of 60 albumin transfusions were performed among a cohort of 5 patients requiring transfusion during the 48 weeks prior to treatment. Following the start of treatment, a single patient required a single albumin transfusion. Additionally, 9 of the 10 patients were hospitalized for a total of 268 in the 48 weeks prior to treatment. Following the start of treatment, 2 patients were hospitalized for a total of 7 days.¹

When assessing safety, results of the study suggested the most common adverse reactions occurring among 2 or more patients included were upper respiratory tract infection, fracture, urticaria, and alopecia.¹

“As the first-ever treatment for CHAPLE, Veopoz is a testament to our commitment to uncovering genetic insights and applying them to the development of effective treatments for patients in need – regardless of the prevalence of their disease,” said George D. Yancopoulos, MD, PhD, board cochair, president, and chief scientific officer at Regeneron.¹ “Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential.”

Regeneron pointed out information on treatment, insurance coverage, and financial support, will be available through the company’s myRARE™ patient support program. Additionally,­­­ the company noted, with the approval of pozelimab-bbfg, the preapproval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed and FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.¹

References:

1. VeopozTM (Pozelimab-bbfg) receives FDA approval as the first treatment for children and adults with Chaple disease. Regeneron Pharmaceuticals Inc. August 18, 2023. Accessed August 18, 2023. https://investor.regeneron.com/news-releases/news-release-details/veopoztm-pozelimab-bbfg-receives-fda-approval-first-treatment.
2. Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease). ClinicalTrials.gov identifier: 04209634. Updated July 12, 2023. Accessed August 18, 2023. https://www.clinicaltrials.gov/ct2/show/NCT004209634