May 31, 2018
The FDA has approved orphan drugs for treatment of X-linked hypophosphatemia, polycystic kidney disease, and cystic fibrosis. This includes Crysvita an immunoglobulin shot that improves the kidneys ability to absorb phosphate in people with X-linked hypophosphatemia. Jynarque is a new drug that slows the decline in kidney function in people with rapidly worsening polycystic kidney disease, and Symdecko is the newest approved orphan product for people with certain cystic fibrosis gene mutations. Symdecko improves the passing of chloride ions in and out of cells to help balance salt and water in the lungs. You can find information on these drugs on the condition pages, as well as a full list of FDA approved drugs with orphan drug designations and approvals on our Web site.
Autosomal dominant polycystic kidney disease
- Not a rare disease