This section highlights FDA-related milestones and regulatory updates for drugs developed by ProQR Therapeutics (PRQR).
Over the past two years, ProQR Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AX-0810 and Axiomer. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
AX-0810 - FDA Regulatory Timeline and Events
AX-0810 is a drug developed by ProQR Therapeutics for the following indication: For cholestatic diseases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AX-0810
- Announced Date:
- June 26, 2025
- Indication:
- For cholestatic diseases
Announcement
ProQR Therapeutics NV announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells.
AI Summary
ProQR Therapeutics NV has taken a major step forward by submitting a Clinical Trial Application (CTA) to the European Medicines Agency (EMA). This move is aimed at starting its first Phase 1 clinical trial for the investigational drug AX-0810. The treatment targets NTCP, a protein found in liver cells that plays a key role in transporting bile acids into the cells. By modulating NTCP, AX-0810 is designed to help reduce the toxic buildup of bile acids, which may lower the risk of liver inflammation, fibrosis, and even liver failure in patients with cholestatic liver diseases.
The Phase 1 trial will focus on evaluating the safety, tolerability, pharmacokinetics, and early signs of target engagement in healthy volunteers. Initial data from the study are expected by Q4 2025.
Read Announcement- Drug:
- AX-0810
- Announced Date:
- June 18, 2024
- Indication:
- For cholestatic diseases
Announcement
ProQR Therapeutics NV announced presentation will detail AX-0810, the Company's program targeting NTCP for cholestatic diseases, including further assessment in in vivo models of biomarkers for NTCP.
AI Summary
ProQR Therapeutics announced that Gerard Platenburg, the company’s Chief Scientific Officer and Co-Founder, will present at the upcoming RNA Editing Summit in Boston. His talk will cover the details of ProQR’s innovative Axiomer RNA editing technology and include a focus on the AX-0810 program. AX-0810 targets the NTCP protein in patients with cholestatic diseases. The presentation will also explain the company’s approach to evaluating biomarkers for NTCP in in vivo models. Through this work, ProQR aims to better understand the potential use of RNA editing to treat disorders linked to this target while advancing its pipeline of therapies. The results from these studies may pave the way for new treatment options for patients with cholestatic conditions.
Read Announcement
Axiomer - FDA Regulatory Timeline and Events
Axiomer is a drug developed by ProQR Therapeutics for the following indication: Editing Technology Adenosine Deaminase Acting on RNA).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Axiomer
- Announced Date:
- June 18, 2024
- Indication:
- Editing Technology Adenosine Deaminase Acting on RNA)
Announcement
ProQR Therapeutics NV announced it will participate in the RNA Editing Summit, June 18-20, 2024, in Boston, Massachusetts.
AI Summary
ProQR Therapeutics NV announced that it will participate in the RNA Editing Summit, held in Boston, Massachusetts, from June 18-20, 2024. During the event, Chief Scientific Officer and Co-Founder Gerard Platenburg will present on June 20 at 2:00 pm ET. His talk, titled "Developing Axiomer RNA Editing Technology Towards Clinical Development," will explore the company’s innovative Axiomer RNA editing platform. This technology uses the naturally occurring enzyme ADAR to make precise changes to RNA by targeting specific nucleotides. The presentation will also cover ProQR’s AX-0810 program, which targets NTCP for cholestatic diseases, demonstrating the potential for this approach to correct mutations and improve therapeutic outcomes. More details about the presentation and the company’s advancements will be available on ProQR’s website.
Read Announcement
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