FDA Approves Regeneron's Drug For Ultra Rare Immune Disorder

The FDA approved  Regeneron Pharmaceuticals Inc's REGN Veopoz (pozelimab-bbfg) for adult and pediatric patients one year of age and older with CHAPLE disease, known as CD55-deficient protein-losing enteropathy. 

Veopoz is the first and only treatment indicated specifically for CHAPLE. 

CHAPLE is an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. In healthy individuals, the complement system is a mechanism for destroying microbes. 

However, those living with CHAPLE cannot regulate complement activity due to mutations in their CD55 gene. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of circulating proteins. There are fewer than ten patients with CHAPLE disease identified in the U.S.

Veopoz, a fully human monoclonal antibody, is designed to target complement factor C5, a protein involved in complement system activation.

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.

Veopoz will be sold in the U.S. at a list price of $34,615.38 per single-use vial, the company told Reuters in an emailed response.

Price Action: REGN shares are up 1.45% at $824.20 on the last check Monday.