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2023-08-28| R&D

Introducing Veopoz™: A Breakthrough Treatment for CHAPLE Disease

by GeneOnline
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Regeneron Pharmaceuticals announces the FDA approval of VEOPOZ, the first treatment for both children and adults dealing with CHAPLE disease, also referred to as CD55-deficient protein-losing enteropathy.

A New Era of Hope

CHAPLE disease, a rare and life-threatening immune disorder, has found its match in Veopoz™. This remarkable achievement marks the first and only treatment tailored specifically for CHAPLE, giving patients and their families newfound hope. Regeneron Pharmaceuticals, Inc. has tirelessly worked to address the pressing medical need, and Veopoz™ stands as a testament to their dedication. 

Dr. Michael Lenardo, Chief of the Molecular Development of the Immune System Section and Co-Director of the Clinical Genomics Program at the National Institute of Allergy and Infectious Disease (NIAID), was a vital participant in the groundbreaking trial. His first-hand experience witnessing the transformative effects of pozelimab on CHAPLE sufferers speaks volumes about the potential of this treatment.

Unraveling the Mystery of CHAPLE

CHAPLE disease, a hereditary immune condition, arises from the overactivation of the complement system – a natural defense mechanism against microbes. Those afflicted by CHAPLE, however, struggle to regulate this system due to mutations in their CD55 gene. This deficiency leads to the immune system attacking healthy cells, causing damage to crucial blood and lymph vessels in the upper digestive tract, along with the loss of vital proteins. With fewer than 10 known CHAPLE patients in the U.S., this disorder is exceedingly rare and debilitating.

Enter Veopoz™, a fully human monoclonal antibody that focuses on complement factor C5 – a key player in the complement system. By targeting this protein, Veopoz™ aims to rein in the excessive complement activity seen in CHAPLE patients, offering them a chance at a better quality of life.

A Note on Safety

While Veopoz™ brings hope, it’s essential to be aware of potential risks. Patients need to complete or update meningococcal vaccination at least two weeks before beginning Veopoz™ treatment. This precaution is vital to prevent potentially life-threatening meningococcal infections.

Regeneron 

Regeneron is unwavering in its mission to bring effective treatments to individuals with rare diseases, such as CHAPLE. Through the myRARE™ patient support program, eligible patients will receive assistance in navigating their treatment journey, from information about the therapy to insurance coverage and financial support.

The approval of Veopoz™ opens the door to exploring its potential in treating other complement-mediated diseases. Regeneron’s research is focused on finding solutions that make a difference in patients’ lives, regardless of the rarity of their conditions.

A Beacon of Progress

Veopoz™’s FDA approval is a significant leap forward, backed by a Phase 2/3 trial that showcased its effectiveness and safety. Patients aged 3 to 19 witnessed normalization of essential protein levels and sustained improvements throughout treatment.

With Veopoz™ as a beacon of progress, those affected by CHAPLE disease can look forward to brighter days ahead. Regeneron Pharmaceuticals, Inc. has taken a giant stride towards improving lives and ushering in a new era of medical possibilities.

 

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