USFDA grants appeal for Ardelyx XPHOZAH

In the response letter, OND directed the FDA's Division of Cardiology and Nephrology (DCN) to work with Ardelyx to develop an appropriate label.

"Ardelyx believes that a label could reflect an indication for patients whose hyperphosphatemia is insufficiently managed on binder therapy," the company stated in a recent release.

In addition, the letter guided Ardelyx to request a meeting with the DCN to determine specific information that will form the basis for resubmission of the NDA for XPHOZAH. Ardelyx will request this meeting as soon as possible to enable the company to resubmit the NDA in the first half of 2023.

"By granting the appeal, we believe that OND has sent a powerful message regarding the importance of bringing innovation to the more than 400,000 patients on dialysis who struggle every day, at every meal, to control their phosphorus levels and yet are unable to do so. For more than 60 years, the only choice physicians had for patients has been phosphate binders, and the patients have desperately needed novel mechanism therapies. This is a momentous day for Ardelyx and for all the members of the broader kidney disease community who have supported the development of XPHOZAH over the past ten years," said Mike Raab, president, and chief executive officer of Ardelyx. "We appreciate the FDA's responsiveness to the clear guidance from the Cardiovascular and Renal Drugs Advisory Committee, recognizing the importance of providing a novel therapy for physicians. This could not have been accomplished without the support from members of the kidney community who spoke at the Advisory Committee meeting about the significant unmet patient need and the important role XPHOZAH could play in the hyperphosphatemia treatment paradigm. We look forward to working with the FDA to bring this important medicine to patients and their treating physicians."

Kevin Martin, M.D., Professor of Internal Medicine in the Division of Nephrology at St. Louis University, added, "This FDA decision is extremely positive for the kidney community and brings us one step closer to having a much-needed novel therapy for the treatment of hyperphosphatemia. The majority of patients are in need of a different approach, as today, despite best efforts with phosphate binders, close to 80% of patients are unable to consistently achieve guideline-established target serum phosphate levels. In clinical trials, XPHOZAH, with its unique mechanism of action that blocks intestinal phosphate transport, provided clinically meaningful reductions in serum phosphate with one small pill twice a day. As was clear during last month's Advisory Committee meeting, patients and physicians are anxious to have access to XPHOZAH."

The OND's decision follows a favorable outcome of the November 16, 2022CRDAC meeting, where the Advisory Committee voted nine to four that the benefits of treatment with XPHOZAH outweigh its risks for the control of serum phosphorus in adults with chronic kidney disease (CKD) on dialysis when administered as a monotherapy, and voted ten to two, with one abstention, that the benefits of treatment with XPHOZAH in combination with phosphate binder treatment outweigh its risks. The NDA for XPHOZAH for the control of serum phosphorus is supported by a comprehensive development program involving more than 1,200 patients and included three Phase 3 clinical trials, all of which met their primary and key secondary endpoints. "Upon approval, XPHOZAH would be the first and only phosphate absorption inhibitor for adult patients with CKD on dialysis with hyperphosphatemia," the company added.