FDA Approves VEOPOZ on August 18th 2023: A New Treatment for CHAPLE Disease

Jul 22, 2024

What is VEOPOZ prescribed for?  

VEOPOZ is prescribed to treat patients 1 year old and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.

What is the name of the drug and what does it do?

The drug, VEOPOZ, pronounced VEE-oh-poz, (generic name: pozelimab-bbfg)is an innovative treatment recently approved by the FDA to manage CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. 

This condition primarily affects children and can lead to severe protein loss, resulting in malnutrition and immune system issues. 

VEOPOZ works by stabilizing the protein levels in patients, providing a significant improvement in their quality of life.

How does it work?

VEOPOZ calms down an overly active part of the immune system called the complement system, which is a problem in patients with CHAPLE disease. 

It specifically stops a protein in this system, called C5, from breaking into smaller pieces that cause inflammation and damage cells. 

By preventing this breakdown, VEOPOZ helps to stop too much protein from being lost through the digestive system. This action helps control the symptoms of CHAPLE disease.

What did the research discover?

Clinical trials have shown that VEOPOZ is effective in increasing serum albumin levels (a major protein in blood plasma) and improving overall clinical outcomes in patients with CD55-deficient PLE. 

The trials noted significant improvements in protein levels and a reduction in the symptoms related to PLE, such as abdominal pain and swelling.

What are some of the side effects?

The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.

  • Upper respiratory tract infection
  • Fractures
  • Urticaria (hives)
  • Alopecia (hair loss)

What are the dosage recommendations and how is it prescribed?

VEOPOZ is administered initially as a 30 mg/kg dose by intravenous infusion, followed by a maintenance dose of 10 mg/kg given as a subcutaneous injection once a week. 

Depending on the clinical response, the maintenance dose may be adjusted. It’s crucial for patients to follow their healthcare provider’s instructions regarding dosing and administration to ensure the best outcomes from the treatment.

Source:

Highlights of Prescribing Information. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761339s000lbl.pdf. Accessed 16 July 2024.

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