FDA Approves Breakthrough Treatments for Ultra-Rare Blood and Immune System Diseases
The U.S. Food and Drug Administration (FDA) has approved Regeneron Pharmaceuticals' drug, Veopoz (pozelimab), for the treatment of CHAPLE disease, a rare blood disease. Veopoz is the first FDA-approved treatment for this life-threatening condition and will be available in the U.S. in the third quarter of this year. CHAPLE disease affects fewer than 10 patients in the U.S. and less than 100 worldwide. However, the drug comes with a boxed warning for serious meningococcal infections. Additionally, Regeneron stated that the approval of Veopoz has resolved pre-approval inspection issues related to the higher dose of its blockbuster eye disease drug, Eylea (aflibercept). The FDA's decision on the 8mg dose of Eylea is expected soon, which could help preserve Regeneron's Eylea franchise and future revenues.
- US FDA approves Regeneron's ultra-rare blood disease drug Reuters
- FDA approves pozelimab-bbfg as first CHAPLE disease treatment for children, adults Contemporary Pediatrics
- Ultra-Rare Immune System Disease Gets First FDA-Approved Treatment Medpage Today
- FDA Approves Palovarotene for Fibrodysplasia Ossificans Progressiva MD Magazine
- FDA approves palovarotene capsules to treat fibrodysplasia ossificans progressiva Contemporary Pediatrics
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