ICYMI: More Steps Required for Experimental ADPKD Drug in Order to Obtain Orphan Designation

According to a story from thenewsreports.com, the biopharmaceutical company XORTX Therapeutics, Inc., recently announced that the company and its partner Cato Clinical Research have received correspondence from the US Food and Drug Administration (FDA) in regards to a recently submitted application for Orphan Drug designation. This application is for the company’s investigational drug XRx-008 as a therapy for autosomal dominant polycystic kidney disease (ADPKD). 

About Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal monogenic human disorder. A monogenic disorder is one which is caused by only a single mutation. It can vary somewhat in presentation, but it is always characterized by cysts on the kidneys, as well as cysts affecting other organs. The disorder is caused by a mutation affecting either PKD1 (85 percent of cases) or PKD2 (15 percent). Those with PKD1 mutations see declines in kidney function more quickly. Dysfunction may not appear until age 40 or 50, but cysts begin to appear early in life, perhaps even prior to birth. Other symptoms include anemia, uremia, hypertension, acute loin pain, liver cysts, and berry aneurysms. There are a number of treatments that can temporarily slow disease progression or relieve symptoms, but many patients will eventually need dialysis or a kidney transplant to survive. To learn more about ADPKD, click here.

The response from the FDA laid out the necessary steps that the company would need to take before XRx-008 can obtain Orphan Drug designation. These steps include:

  1. Manufacture XRx-008 that is ready for clinical trial use.
  2. Respond and finish Investigational New Drug Application (IND) filing with the agency. 
  3. Describe improved bioavailability in humans.
  4. Submit protocol for Phase 2/3 trials, discuss the approval of special protocol with FDA representatives, and begin the first clinical trial of XRx-008.

About XRx-008

The company aims to have its Phase 2/3 clinical trials ready in the year 2019. XRx-008 aims to slow the progression of ADPKD and maintain kidney function by regulating the concentration of uric acid, which has been implicated in disease progression in earlier research. 

Orphan Drug designation is usually limited to therapies intended to treat rare diseases, which is defined as diseases that affect less than 200,000 people in the US. To qualify, a drug must either offer advantages in effectiveness and/or safety over current therapies or fulfill a currently unmet medical need.


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