Regeneron Receives FDA Approval for Veopoz™

Regeneron, Inc. (Nasdaq: REGN) has received FDA approval for Veopoz™ (pozelimab-bbfg) as a treatment for children and adults with CHAPLE disease, an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms. Veopoz is the first and only treatment indicated specifically for CHAPLE disease and represents the 10th FDA-approved medicine invented by Regeneron.

There are fewer than 10 patients with CHAPLE disease identified in the U.S.

George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron, said, “As the first-ever treatment for CHAPLE, Veopoz is a testament to our commitment to uncovering genetic insights and applying them to the development of effective treatments for patients in need – regardless of the prevalence of their disease. Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential.”


Veopoz was reviewed under Priority Review, and the Company received a Rare Pediatric Disease Priority Review voucher upon approval. Veopoz was previously granted Rare Pediatric Disease designation, Orphan Disease designation and Fast Track designation.

The FDA approval is based on results from a Phase 2/3 open-label trial that investigated the efficacy and safety of pozelimab in 10 patients aged 3 to 19 (median of 8.5 years).

All ten patients achieved normalization of serum albumin and serum IgG concentrations by week 12 and maintained these concentrations through at least 72 weeks of treatment.

About Regeneron

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center®, which is conducting one of the largest genetics sequencing efforts in the world.

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Regeneron Receives FDA Approval for Veopoz™

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