Regeneron wins approval for rare immune disease therapy pozelimab
Olivier Le Moal

The US FDA has approved Regeneron Pharmaceuticals' (NASDAQ:REGN) pozelimab for treatment of the rare immune disease CHAPLE disease.
The approval is the first specifically for the condition, which is characterized by severe gastrointestinal distress and deep vein blood clots.
Pozelimab's price tag was not disclosed.

Veopoz, the drug's brand name, works by targeting complement factor C5, a protein involved in complement system activation.

Regeneron (REGN) said pozelimab is also under investigation in combination with Alnylam Pharmaceuticals' (ALNY) cemdisiran for the other complement-mediated disorders including paroxysmal nocturnal hemoglobinuria and myasthenia gravis.


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