The US Food and Drug Administration (FDA) has accepted a priority review of the biologics license application (BLA) for Regeneron Pharmaceuticals’ pozelimab to treat children and adults with ultra-rare CHAPLE disease.
Currently, there are no approved treatments for CHAPLE, a life-threatening hereditary immune disease caused by an overactivation of the complement system.
The investigational, fully human monoclonal antibody, Pozelimab has been designed to block the complement factor C5 activity.
C5 is a protein that is involved in the activation of the complement system.
The antibody is developed using the company’s VelocImmune technology.
The regulatory body is expected to decide on 20 August.
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The BLA submitted to the FDA is supported by the data obtained from an open-label Phase II/III trial, designed to evaluate the efficacy and safety of pozelimab in ten patients aged one year or above.
The findings showed that 100% of patients had quick and sustained normalisation of serum albumin (a disease biomarker) and improvement or no worsening of clinical symptoms at 24 weeks.
In the trial, the clinical symptoms were assessed, including the number of bowel movements per day, abdominal pain, and investigator-assessed facial and peripheral edema.
In April 2020, pozelimab was designated as a drug for a “rare pediatric disease” to treat CHAPLE by the US FDA, with Regeneron having an opportunity to get a rare pediatric disease priority review voucher if pozelimab is approved for CHAPLE.
It was also granted Orphan Drug Designation during the same time.
It is also being assessed along with Alnylam’s cemdisiran (siRNAi C5 inhibitor) to treat other complement-mediated disorders, including myasthenia gravis (MG) and paroxysmal nocturnal hemoglobinuria (PNH).
Earlier this month, Regeneron received US FDA approval for its Eylea (aflibercept) injection to treat the retinopathy of prematurity (ROP) in preterm infants.