XORTX Therapeutics Inc. (CNSX:XRX) has achieved a significant milestone in the development of XRx-008. The drug treats autosomal dominant polycystic kidney disease (PKD). The company has received a response letter from the U.S Food and Drug Administration (FDA).
XRx-008 Orphan Drug Designation
The FDA’s letter is in response to a recently submitted Orphan Drug Designation application. The bio-pharmaceutical company made the submission mid-September as it provided additional information as requested by the agency.
Further, the company intends to make an additional submission in 2019 as it pursues ODD status for XRs-008. In addition, plans are already underway to complete manufacturing of clinical study. XORTX Therapeutics also intends to make an Investigational New Drug Application with the agency.
XORTX Therapeutics is in the process of submitting Pivotal phase 2/3 protocol for XRx-008. The company also intends to open discussion with the FDA over special protocol approval as it also eyes clinical trial. According to Chief Executive Officer, Dr. Allen Davidoff, XRx-008 holds promise as a meaningful therapy for PKD.
Over the past two quarter, XORTX Therapeutic has focused attention on initiating communications with the FDA. Focus going forward is accelerating its development.
“Feedback and discussions with the FDA provided a clear set of steps to accelerate and focus our priorities, staff, and funding in the future. Over the next year, XORTX will focus on executing on the critical path steps that position the Company for a pivotal phase 2/3 clinical trial in 2019, and we will continue to communicate with the FDA regarding an SPA for that trial,” Davidoff said in a statement.
Advancing XRx-008 into Phase 2/3 pivotal clinical trial is at the top on the agenda. The candidate drug has the potential to address a significant problem afflicting PKD patients. Scientific and clinical evidence suggests that the concentration of uric acid in patients with PKD can somehow accelerate the disease’s progression.
Managing purine and Uric acid is an area of focus with XRx-008. It reportedly will go a long way in reducing the rate at which the kidney slows down when it comes to filtering. While there is one FDA approved therapy for the disease, it has turned out to be ineffective in its mandate.
Accordinly, achieving Orphan Drug Designation for XRx-008 would be a significant milestone. The designation targets treatments for medical conditions that affect fewer than 200,000 patients living in the U.S. However, the designation does not indicate that a drug is safe, effective or legal to manufacture in the country
Instead, the designation gives companies access to a number of benefits from the federal government. For instance, XORTX Therapeutics upon ODD designation would be able to enjoy reduced taxes as well as grants to accelerate development of the candidate drug.