Regeneron Pharmaceuticals’ Veopoz (pozelimab-bbfg) has been approved by the US Food and Drug Administration (FDA) to treat patients with an ultra-rare immune disease.
The human monoclonal antibody has been specifically authorised for use in adult and paediatric patients aged one year and older with CHAPLE disease.
Fewer than ten patients in the US have been diagnosed with the condition, which is also known as CD55-deficient protein-losing enteropathy.
People with CHAPLE have a mutation in their CD55 gene, which regulates the body’s mechanism for destroying microbes. Without proper regulation, the mechanism may start attacking normal cells in the body.
Veopoz is now the first treatment indicated specifically for CHAPLE and is designed to target complement factor C5, a protein involved in complement system activation.
The FDA’s decision is supported by results from a phase 2/3 trial that evaluated the safety and efficacy of the drug in ten patients aged three to 19 years, who were given a single loading dose of Veopoz intravenously on day one, followed by subcutaneous weekly weight-based doses.
All ten patients achieved normalisation of serum albumin and serum IgG concentrations by week 12, the company said, and maintained those concentrations through at least 72 weeks of treatment.
“As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that [Veopoz] achieves in those suffering from CHAPLE,” said Michael Lenardo, chief of the molecular development and immune system section and the founder and co-director of the clinical genomics programme at the National Institute of Allergy and Infectious Disease, National Institutes of Health.
As part of its ongoing development programme, Veopoz is also being evaluated in combination with Alnylam’s cemdisiran to treat other complement-mediated disorders, including paroxysmal nocturnal haemoglobinuria and myasthenia gravis.
George Yancopoulos, Regeneron’s president and chief scientific officer, said: “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.
“Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programmes to explore its broader potential.”