The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Ardelyx’s XPHOZAH (tenapanor) for the treatment of pediatric hyperphosphatemia.
XPHOZAH is a single tablet taken twice daily that offers a first-in-class mechanism of action that blocks phosphate absorption through its primary pathway.
“We are pleased that the FDA has recognized the important need among pediatric patients with hyperphosphatemia for treatment options,” said Mike Raab, president and chief executive officer of Ardelyx. “This designation highlights the importance of evaluating treatment options for all patients with hyperphosphatemia, and we look forward to sharing more details on this important designation by FDA in the future.”
The FDA’s Orphan Drug Designation supports the development and evaluation of new drugs or biological products to prevent, diagnose or treat rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug benefits can include potential market exclusivity for seven years after approval, exemption from user fees and tax credits for qualified clinical trials.
In October, XPHOZAH was approved by the FDA to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis as add-on therapy in patients who have an inadequate response to phosphate binders or who are intolerant of any dose of phosphate binder therapy.