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.
Select a button below to view the list of FDA events for that drug.
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 25, 2026
- Indication:
- For cholestatic diseases
Announcement
ProQR Therapeutics N.V announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810, the Company’s first investigational Axiomer RNA editing oligonucleotide (EON), in healthy volunteers.
AI Summary
ProQR Therapeutics said its Phase 1 study of AX-0810, its first Axiomer RNA editing oligonucleotide, showed positive target engagement in healthy volunteers. AX-0810 is given under the skin and is designed to block NTCP, a liver transporter that helps move bile acids from the blood into the liver. By lowering NTCP activity, the treatment aims to reduce harmful bile acid buildup, which is linked to cholestatic liver diseases such as biliary atresia.
In the study, 33 healthy volunteers were enrolled, and data from the 3 mg/kg and 6 mg/kg groups showed dose-dependent increases in urinary bile acid excretion, confirming the drug hit its target. The company also reported a favorable safety profile, with no serious side effects or itching so far. ProQR plans to share full Phase 1 results later this year and is moving toward Phase 2 development in biliary atresia.
Read Announcement- Drug:
- AX-0810
- Announced Date:
- January 8, 2026
- Indication:
- For cholestatic diseases
Announcement
ProQR Therapeutics N.V announced encouraging initial safety and PK data from the first cohort of healthy volunteers in its ongoing Phase 1 trial of AX-0810 and provided an update on broader pipeline progress and anticipated 2026 milestones.
AI Summary
ProQR reported encouraging initial safety and pharmacokinetic (PK) data from the first cohort of its Phase 1 AX-0810 trial in healthy volunteers. After four weeks of dosing (3 mg/kg cohort) there were no serious adverse events or clinically meaningful lab changes, and PK matched non‑clinical expectations. The randomized, double‑blind, placebo‑controlled, multiple‑dose study will enroll up to 33 participants who receive four weekly subcutaneous injections and a 12‑week safety follow‑up. Enrollment and dosing are ongoing, and ProQR expects target‑engagement biomarker data in H1 2026, with a patient cohort to follow.
ProQR also selected development candidates for AX‑2402 (Rett syndrome, MECP2 R270X) and AX‑2911 (MASH, PNPLA3 I148M), reporting promising preclinical proof‑of‑concept results that support moving toward human studies.
The company noted progress in its Eli Lilly collaboration, which generated $4.5 million in 2025, and said the partnership and milestones help strengthen its cash runway into mid‑2027. Key 2026 priorities include reporting AX‑0810 target engagement, initiating the patient cohort, advancing AX‑2402 toward first‑in‑human trials, and sharing more preclinical data.
Read Announcement- Drug:
- AX-0810
- Announced Date:
- October 20, 2025
- Indication:
- For cholestatic diseases
Announcement
ProQR Therapeutics N.V. announced that following review under the new European Medicines Agency (EMA) centralized review process, the Central Committee on Research Involving Human Subjects (CCMO) has authorized ProQR's Clinical Trial Application (CTA) for a Phase 1 study of AX-0810 in healthy volunteers. AX-0810 is the Company's lead investigational editing oligonucleotide (EON) targeting NTCP, which is being developed for the treatment of cholestatic diseases like primary sclerosing cholangitis and biliary atresia.
AI Summary
ProQR Therapeutics N.V. announced that the Central Committee on Research Involving Human Subjects (CCMO) has approved its Clinical Trial Application for AX-0810 through the new EMA centralized review process. AX-0810 is ProQR’s lead investigational editing oligonucleotide targeting NTCP, a key protein involved in bile acid transport. The therapy aims to treat cholestatic liver diseases such as primary sclerosing cholangitis and biliary atresia by reducing bile acid accumulation and preventing liver damage.
With this authorization, ProQR can begin dosing healthy volunteers in a Phase 1 study in the Netherlands. The trial will assess safety, tolerability, pharmacokinetics, and pharmacodynamics using biomarker measurements to confirm target engagement. AX-0810 is the first Axiomer program to enter clinical development and could offer a disease-modifying approach for patients with unmet needs. Positive results in this study will guide future clinical development of AX-0810 in patients with cholestatic diseases.
Read Announcement- 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
Axiomer FDA Regulatory 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:
- May 11, 2026
- Indication:
- Editing Technology Adenosine Deaminase Acting on RNA)
Announcement
ProQR Therapeutics N.V announced presentations at several upcoming scientific conferences, highlighting continued progress in the application of its Axiomer RNA editing platform..
AI Summary
ProQR Therapeutics announced presentations at upcoming conferences—TIDES USA, ASGCT, RNA Society, and EASL—highlighting continued progress of its Axiomer RNA editing platform. Sessions will cover broad applicability of ADAR RNA editing, Axiomer‑mediated correction of a premature stop codon in MECP2 for Rett syndrome, and conversion of the PNPLA3 I148M variant toward a wild‑type‑like form to reduce hepatic steatosis in MASLD/MASH.
At the RNA Society meeting ProQR will present an oligonucleotide design platform for ADAR‑mediated editing (ID# 625). The poster reports Axiomer advances that optimize the intracellular journey: engineered oligonucleotides (EONs) achieve efficient cellular uptake and show measurable endosomal escape in vitro, enabling productive access to the nucleus required for editing. Presenter: Nandan Varadarajan.
Presentation slides and posters will be made available in the Publications and Presentations section of ProQR’s website at www.proqr.com.
Read Announcement- Drug:
- Axiomer
- Announced Date:
- July 28, 2025
- Indication:
- Editing Technology Adenosine Deaminase Acting on RNA)
Announcement
ProQR Therapeutics NV announced that it will participate in the RNA Editing Summit taking place July 29-31, 2025, in Boston, MA.
Read Announcement
