FDA approves Veopoz for CHAPLE disease
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, according to a press release. This is the first and only treatment indicated specifically for CHAPLE.
FDA approval is based on a Phase 2/3 trial involving 10 patients aged 3 to 19 (average age of 8.5 years). Patients received an initial intravenous loading dose of pozelimab at 30 mg/kg on day 1, followed by weekly subcutaneous doses based on their weight.
Results showed that all 1- patients achieved normal levels of serum albumin and serum IgG by week 12 and maintained these levels for at least 72 weeks of treatment. Prior to treatment, 5 out of the 10 patients needed 60 albumin transfusions over 48 weeks. After starting pozelimab, only 1 patient needed 1 albumin transfusion over the same period. Prior to treatment, 9 of the 10 patients were hospitalized for a combined total of 268 days over 48 weeks. After treatment initiation, only 2 patients were hospitalized for a total of 7 days during the same 48-week period.
“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, Molecular Development of the Immune System Section; Co-Director, Clinical Genomics Program, National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH) in a press release. “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.”
Read the full press release here.