XORTX Therapeutics Inc. (NASDAQ: XRTX) is moving forward with its XRx-026 program for the treatment of gout, announcing that it has requested a Type C meeting with the U.S. Food and Drug Administration (FDA). The meeting is intended to facilitate a review of the program's readiness for a New Drug Application (NDA) submission, potentially paving the way for marketing approval of XORLO™ in the United States.
The XRx-026 program is centered around XORLO™, XORTX's proprietary formulation of oxypurinol. This drug is specifically targeted at individuals with gout who are intolerant to allopurinol, a first-line treatment. Allopurinol intolerance affects 3 to 5% of patients, creating an unmet medical need that XORTX aims to address.
Addressing Allopurinol Intolerance in Gout
Gout, an inflammatory arthritis triggered by uric acid crystallization in the joints, affects approximately 9.2 million people in the U.S. While xanthine oxidase inhibitors (XOIs) like allopurinol are commonly prescribed to lower uric acid levels, intolerance can be a significant barrier. Febuxostat, another XOI, has faced challenges due to a Black Box warning related to cardiovascular risks, further underscoring the need for alternative treatments.
Dr. Allen Davidoff, CEO of XORTX, emphasized the importance of this program, stating, "The key elements of the XRx-026 program have advanced sufficiently to warrant a robust program review with the FDA... The prospect of helping individuals with gout that have otherwise few therapeutic options... is compelling."
Regulatory Pathway and Market Opportunity
XORTX is pursuing the FDA's 505(b)(2) development pathway, which could expedite the approval process by leveraging existing data on oxypurinol. The company anticipates the Type C meeting with the FDA will occur within 75 days of the request. Furthermore, XORTX intends to seek FDA orphan drug designation (ODD) for the XRx-026 program, a status previously granted to oxypurinol for allopurinol-intolerant gout.
The addressable market for gout treatments is substantial. In North America, approximately 3.5 million people suffer from gout due to elevated uric acid levels. The limitations and risks associated with existing treatments like febuxostat have opened a door for novel XOIs like XORLO™ to fill the unmet need.
XORTX's Broader Pipeline
XORTX is also developing XRx-008 for ADPKD and XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections, as well as XRx-225, a pre-clinical stage program for Type 2 Diabetic Nephropathy. XORTX is dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases.
