FDA approves Veopoz as first treatment for children and adults with CHAPLE disease
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Key takeaways:
- CHAPLE disease is a rare hereditary disease that involves overactivation of the complement system.
- Veopoz, which targets complement factor C5, is approved for adults and children aged 1 year and older.
The FDA has approved the use of Veopoz for treatment of adults and children aged 1 year and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy.
According to a Regeneron press release, Veopoz (pozelimab-bbfg, Regeneron) is a fully human, monoclonal antibody designed to target complement factor C5, a protein involved in the activation of the complement system. It is the first-ever treatment for CHAPLE, an ultra-rare hereditary immune disease that has been identified in fewer than 10 patients in the United States.
Patients with CHAPLE cannot regulate complement activity because of mutations in their CD55 gene. Without regulation, the complement system can then attack normal cells and damage blood and lymph vessels in the upper digestive tract, causing loss of circulating proteins.
“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” Michael Lenardo, MD, chief of molecular development of the immune system section and co-director of the clinical genomics program at the National Institute of Allergy and Infectious Disease, said in the release.
The FDA based its approval on the results of a phase 2/3 open-label trial involving 10 patients aged 3 to 19 years (median, 8.5 years). Participants received a single loading dose of IV pozelimab 30 mg/kg on day one and subsequent weight-based, subcutaneous weekly doses.
According to the release, all participants achieved normalization of serum albumin and serum IgE concentrations by week 12, which were maintained through at least week 72. Five patients received 60 albumin transfusions in the 48 weeks before treatment with pozelimab, and one patient received an albumin transfusion in the 48 weeks after treatment.
Nine patients were hospitalized for a total of 268 days in the 48 weeks before treatment, and two were hospitalized for a total of 7 days in the 48 weeks after starting treatment. Upper respiratory tract infection, fracture, urticaria and alopecia were the most common adverse reactions occurring in two or more patients, the release stated.
Noting that life-threatening or fatal meningococcal infections have occurred in patients treated with complement inhibitors, Regeneron recommends complete or updated meningococcal vaccination at least 2 weeks before the first dose of pozelimab.
“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,” George D. Yancopoulos, MD, PhD, president and chief scientific officer at Regeneron, said in the release.
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