Ardelyx, Inc. (Nasdaq: ARDX) is a biopharmaceutical company dedicated to discovering, developing, and commercializing innovative medicines to address unmet medical needs. The company recently announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to XPHOZAH® (tenapanor) for treating pediatric hyperphosphatemia. XPHOZAH, a first-in-class phosphate absorption inhibitor taken twice daily, works by blocking phosphate absorption through its primary pathway.

Ardelyx, expressed satisfaction with the FDA's recognition of the need for treatment options for pediatric patients with hyperphosphatemia. He emphasized the significance of evaluating treatment options for all patients with hyperphosphatemia and anticipates sharing more details on the FDA designation in the future.

Orphan Drug Designation from the FDA facilitates the development of drugs for rare diseases affecting fewer than 200,000 people in the U.S. Benefits include potential market exclusivity for seven years post-approval, exemption from user fees, and tax credits for qualified clinical trials.

In October, XPHOZAH received FDA approval for reducing serum phosphorus in adults with chronic kidney disease (CKD) on dialysis, serving as add-on therapy for patients with an inadequate response to phosphate binders or those intolerant to any dose of phosphate binder therapy.

XPHOZAH, discovered and developed by Ardelyx, acts as a phosphate absorption inhibitor, targeting the sodium hydrogen exchanger 3 (NHE3) locally in the gut. It is taken twice daily as a single tablet, with diarrhea being the most common side effect reported in clinical trials.

Hyperphosphatemia, characterized by elevated blood phosphate levels, is a serious condition affecting a significant portion of the 550,000 CKD patients on maintenance dialysis in the U.S. As kidney function declines, the body's ability to eliminate excess phosphate diminishes, making hyperphosphatemia nearly universal in this patient population.

XPHOZAH includes contraindications in pediatric patients under 6 years of age and those with known or suspected mechanical gastrointestinal obstruction. Patients may experience severe diarrhea, necessitating discontinuation of XPHOZAH in such cases. The most common adverse reaction reported is diarrhea, with the majority being mild-to-moderate in severity and resolving over time or with dose reduction.

XPHOZAH is indicated to reduce serum phosphorus in adults with CKD on dialysis as add-on therapy for those with an inadequate response to phosphate binders or intolerance to any dose of phosphate binder therapy. The recommended dosage is 30 mg BID.

 

Source: globenewswire.com

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