XPHOZAH® (tenapanor) Granted Orphan Drug Designation by U.S. FDA for the Treatment of Pediatric Hyperphosphatemia

Rhea-AI Summary

Ardelyx, Inc. (Nasdaq: ARDX) announced that the FDA granted Orphan Drug Designation to XPHOZAH® (tenapanor) for pediatric hyperphosphatemia. XPHOZAH is a first-in-class medicine that blocks phosphate absorption. The designation supports the development of new drugs for rare diseases, providing potential market exclusivity for seven years after approval, exemption from user fees, and tax credits for qualified clinical trials.

WALTHAM, Mass., Nov. 15, 2023 (GLOBE NEWSWIRE) -- Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to 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.

About XPHOZAH^® (tenapanor)
XPHOZAH, discovered and developed by Ardelyx, is a first-in-class, phosphate absorption inhibitor with a differentiated mechanism of action that acts locally in the gut to inhibit the sodium hydrogen exchanger 3 (NHE3), thereby reducing phosphate absorption through the paracellular pathway, the primary pathway of phosphate absorption. XPHOZAH is a single tablet, taken twice daily. Diarrhea was the most common side effect experienced by patients taking XPHOZAH in clinical trials. Please see additional full Prescribing Information.

About Hyperphosphatemia
Hyperphosphatemia is a serious condition, defined elevated levels of phosphate in the blood, which affects the vast majority of the 550,000 patients in the United States with chronic kidney disease (CKD) on maintenance dialysis. The kidneys are responsible for eliminating excess phosphate and as kidney function declines, phosphate is not adequately eliminated from the body. As a result, hyperphosphatemia is a nearly universal condition among people with CKD on maintenance dialysis, with internationally recognized KDIGO treatment guidelines that recommend lowering elevated phosphate levels toward the normal range (2.5-4.5mg/dL).

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
XPHOZAH is contraindicated in:

• Pediatric patients under 6 years of age
• Patients with known or suspected mechanical gastrointestinal obstruction

WARNINGS AND PRECAUTIONS
Diarrhea
Patients may experience severe diarrhea. Treatment with XPHOZAH should be discontinued in patients who develop severe diarrhea.

MOST COMMON ADVERSE REACTIONS
Diarrhea, which occurred in 43-53% of patients, was the only adverse reaction reported in at least 5% of XPHOZAH-treated patients with CKD on dialysis across trials. The majority of diarrhea events in the XPHOZAH-treated patients were reported to be mild-to-moderate in severity and resolved over time, or with dose reduction. Diarrhea was typically reported soon after initiation but could occur at any time during treatment with XPHOZAH. Severe diarrhea was reported in 5% of XPHOZAH-treated patients in these trials.

INDICATION
XPHOZAH (tenapanor), 30 mg BID, is indicated 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.

For additional safety information, please see full Prescribing Information.

About Ardelyx, Inc.
Ardelyx was founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs. Ardelyx has two commercial products approved in the United States, IBSRELA^® (tenapanor) and XPHOZAH^® (tenapanor) as well as early-stage pipeline candidates. Ardelyx has agreements for the development and commercialization of tenapanor outside of the U.S. Kyowa Kirin has received approval for PHOZEVEL^® (tenapanor) for hyperphosphatemia in Japan. A New Drug Application for tenapanor for hyperphosphatemia has been submitted in China with Fosun Pharma. Knight Therapeutics commercializes IBSRELA in Canada. For more information, please visit https://ardelyx.com/ and connect with us on X (formerly known as Twitter), LinkedIn and Facebook.

Investor and Media Contacts:
Caitlin Lowie
clowie@ardelyx.com

FAQ

What is the FDA's Orphan Drug Designation for?

The FDA's Orphan Drug Designation is for the treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S.

What is XPHOZAH® (tenapanor)?

XPHOZAH® (tenapanor) is a first-in-class medicine that blocks phosphate absorption and is taken twice daily as a single tablet.

What are the benefits of Orphan Drug Designation?

The benefits include potential market exclusivity for seven years after approval, exemption from user fees, and tax credits for qualified clinical trials.

What was XPHOZAH® (tenapanor) previously approved for?

In October, XPHOZAH® (tenapanor) was approved by the FDA to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis.