Regeneron's C5 Monoclonal Antibody Pozelimab Receives FDA Priority Review for CHAPLE Disease

On February 21, Regeneron announced that the Biologics License Application (BLA) for Pozelimab to treat CHAPLE syndrome received priority review from the FDA, with a PDUFA date of August 20, 2023. Currently, there are no approved therapies available for this disease.

CHAPLE Syndrome, also known as CD55 Deficiency with Complement Hyperactivation, Angiopathic Thrombosis, and Protein-Losing Enteropathy or CD55 Deficiency-Mediated Protein-Losing Enteropathy, is an ultra-rare hereditary immune disorder caused by biallelic loss-of-function mutations in CD55 that lead to excessive complement activation. The lack of CD55 in the human body causes the complement system to attack normal cells, subsequently damaging lymphatic vessels and blood vessels in the upper digestive tract, resulting in the loss of blood cells and proteins. Most patients experience symptoms such as abdominal pain, bloody diarrhea, vomiting, malnutrition, slow growth, leg swelling (edema), recurrent infections, and thrombosis, which can be life-threatening in severe cases. According to statistics, there are fewer than 100 CHAPLE syndrome patients worldwide.

Pozelimab is a fully human IgG4 monoclonal antibody targeting complement factor C5, developed by Regeneron using its proprietary VelocImmune technology platform. It aims to block the activity of complement factor C5 and prevent complement pathway-mediated diseases.

This BLA is primarily based on positive results from a Phase II/III single-arm clinical trial (NCT04209634). The study included 10 patients aged 1 year and older with CHAPLE syndrome, aiming to evaluate the efficacy and safety of Pozelimab. Patients were required to receive an initial intravenous injection of Pozelimab at a dose of 30 mg/kg on Day 1, followed by weekly subcutaneous injections of Pozelimab based on body weight. The primary endpoint was the proportion of patients achieving normalization of serum albumin levels and showing improvement in clinical symptoms (including abdominal pain, daily bowel movements, and facial and peripheral edema).

The results showed that at week 24 of treatment, 100% of patients' serum albumin levels rapidly and continuously returned to normal, with clinical symptoms improved or no deterioration. In addition, the number of hospitalization days and albumin infusion times significantly decreased, and the age-weight score and age-height score increased in a clinically meaningful way. In terms of safety, 7 patients experienced mild or moderate adverse events (AEs), the most common AEs being iron deficiency, fever, rhinitis, urticaria, and vomiting.

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