Pozelimab

Generic Name
Pozelimab
Brand Names
Veopoz
Drug Type
Biotech
Chemical Formula
-
CAS Number
2096328-94-6
Unique Ingredient Identifier
0JJ21K6L2I
Background

CD55-deficient protein-losing enteropathy (PLE), or CHAPLE disease, is an ultra-rare hereditary disease, with fewer than 100 patients diagnosed worldwide or fewer than 10 patients in the US. The pathophysiology of this disease is mainly attributed to the deficiency of the CD55 protein, which is the main regulator of the complement cascade.. Under normal circ...

Indication

Pozelimab is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.

Associated Conditions
CHAPLE disease
Associated Therapies
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drugs.com
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Novel Combination of Pozelimab and Cemdisiran (Poze-Cemdi) Achieved Greater Control of Intravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Compared to Ravulizumab

Pozelimab and cemdisiran combination shows superior inhibition of terminal complement in PNH patients, achieving higher LDH control and normalization rates compared to ravulizumab, with potential for self-administration.
biospace.com
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Regeneron Reports Third Quarter 2024 Financial and Operating Results

Q3 2024 revenues up 11% to $3.72 billion; Dupixent global net sales by Sanofi up 23% to $3.82 billion; U.S. net sales for EYLEA HD and EYLEA up 3% to $1.54 billion; Libtayo global net sales up 24% to $289 million; GAAP diluted EPS up 30% to $11.54; FDA approves Dupixent for COPD and eosinophilic phenotype; positive results for Dupixent in CSU and BP trials.
neurologylive.com
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Phase 3 NIMBLE Trial to Test Effects of Pozelimab and Cemdisiran in Myasthenia Gravis

The NIMBLE phase 3 trial assesses pozelimab and cemdisiran combination therapy in MG patients, with 235 participants. The study includes a 24-week double-blind period, 28-week extension, 68-week open-label, and 52-week off-treatment follow-up. Primary endpoint is change in MG-ADL score. Secondary outcomes include QMG score, MG Composite score, and safety measures. Patients must have MG-ADL scores ≥6, excluding those with MuSK antibodies or thymectomy history.
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