Regeneron Report the US FDA Acceptance of BLA for Priority Review of Pozelimab for the Treatment of Ultra-Rare Chaple Disease
- The US FDA has accepted the BLA for Priority Review of pozelimab for adults & children aged 1yr. with CHAPLE disease. The US FDA’s decision is expected in Aug 2023
- The BLA was based on the P-II/III open-label trial evaluating pozelimab (30mg/kg, IV on day 1), followed by SC, qw doses in 10 patients aged ≥1yr. The trial met its co-primary EPs i.e., rapid & sustained normalization of serum albumin (100%) and improvement or no worsening of clinical symptoms @24wks.
- Clinical symptoms incl. abdominal pain, no. of bowel movements per day, investigator-assessed facial & peripheral edema. The 2EPs showed marked reductions in hospitalization days, total no. of albumin transfusions, and increases in body weight for age & stature for age, no AEs led to treatment discontinuation
Ref: Regeneron | Image: Regeneron
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