Drug updated on 4/16/2024
| Dosage Form | Injection (intravenous or subcutaneous; 400 mg/2 mL [200 mg/mL] in a single-dose vial) |
| Drug Class | Complement inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.
Summary
- Pozelimab-bbfg (Veopoz) 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.
- The information about Veopoz is derived from a single study, which is a randomized controlled trial. This trial was an open-label, single-arm, historically controlled multicentre phase 2 and 3 trial conducted across hospitals in Thailand, Türkiye, and the USA.
- In the study involving ten patients primarily from pediatric populations, aged around eight years old on average, hailing from diverse backgrounds including Türkiye, Syria, Thailand, and Bolivia. An initial intravenous loading dose of 30 mg/kg bodyweight was administered followed by once-per-week subcutaneous doses adjusted based on body weight.
- All ten patients showed serum albumin normalization along with improvement in clinical outcomes such as abdominal pain reduction, bowel movement frequency regulation, and facial/peripheral edema alleviation at week 24 compared to baseline, indicating the efficacy of Veopoz against CHAPLE disease.
- Nine out of ten participants experienced adverse events during the course of treatment, two being severe incidents; one event was considered related to Pozelimab-bbfg (Veopoz). Despite these safety concerns, the benefits offered by this drug could outweigh potential risks, especially considering its unique position as the currently approved therapeutic option for treating an ultra-rare genetic disorder like CHAPLE disease where alternative options are scarce or non-existent.
- Given that there is only one study reviewed, it's essential to note that broader analyses involving comparisons with other treatments or larger, different population types would provide more comprehensive safety profile, effectiveness data regarding Veopoz. However, this preliminary finding suggests a significant advancement in care, prioritizing a previously unmet medical need particularly among younger patient groups suffering from severe protein-losing enteropathy due to overactivation of the complement system caused by CD55 deficiency.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Veopoz (pozelimab-bbfg) Prescribing Information. | 2023 | Regeneron Pharmaceuticals, Tarrytown, NY |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study. | 10Subjects F: 60% M: 40% | 2024 | The Lancet |
Sex Distribution:
F:60%
M:40%
10Subjects
Year:
2024
Source:The Lancet