Pharma Focus America

XORTX Announces Submission of Orphan Drug Designation Request for XRx-008 Program to Treat Autosomal Dominant Kidney Disease

Thursday, February 02, 2023

XORTX Therapeutics Inc., a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce it submitted an Orphan Drug Designation (“ODD”) Request to the US Food and Drug Administration (“FDA”) for the XRx-008 program for the treatment of Autosomal Dominant Polycystic Kidney Disease (“ADPKD”). XORTX anticipates updates regarding the ODD application and will provide announcements as information becomes available.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US) are eligible to apply for ODD, which provides multiple benefits to the sponsor during development and after approval. XORTX intends to pursue these benefits as part of the drug development for XRx-008 for treatment of ADPKD, pending designation of the ODD Request.

Benefits of Orphan Drug Designation

Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 25% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and special assistance to companies that develop drugs for rare patient populations is another benefit of ODD. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

  • Seven years exclusivity post-approval
  • Tax credits of 25% off the clinical drug testing cost awarded upon approval
  • Waiver of FDA User Fees

XORTX recently presented positive results in animal models of PKD in both mouse and rat, showing the ability of XORTX’s proprietary formulation of oxypurinol, XORLOTM, to attenuate expansion of kidneys, due to the cyst promoting effects of aberrant purine metabolism and hyperuricemia - at the American Society of Nephrology meeting during the November 2022 meeting.

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