This section highlights FDA-related milestones and regulatory updates for drugs developed by uniQure (QURE).
Over the past two years, uniQure has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AMT-130, AMT-162, AMT-191, and AMT-260. 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.
AMT-130 FDA Regulatory Timeline and Events
AMT-130 is a drug developed by uniQure for the following indication: Huntington's Disease.
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:
- AMT-130
- Announced Date:
- June 2, 2025
- Indication:
- Huntington's Disease
Announcement
uniQure N.V. today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington's disease.
AI Summary
uniQure N.V. provided an update on its investigational gene therapy, AMT-130, for Huntington’s disease. The company has reached alignment with the FDA on key elements needed for a Biologics License Application (BLA) submission. This agreement covers important parts of the statistical analysis plan (SAP) and the Chemistry, Manufacturing and Controls (CMC) information. The planned BLA submission is set for the first quarter of 2026, and the company is targeting an accelerated approval pathway based on several years of clinical data.
uniQure’s progress includes plans to share three-year top-line data by the third quarter of 2025. This update marks a significant step toward pursuing the first disease-modifying treatment for Huntington’s disease, demonstrating the company’s commitment to addressing the urgent medical needs associated with this severe condition.
Read Announcement- Drug:
- AMT-130
- Announced Date:
- April 17, 2025
- Indication:
- Huntington's Disease
Announcement
uniQure N.V announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available.
AI Summary
uniQure N.V. announced that the FDA has granted Breakthrough Therapy designation to its gene therapy AMT-130 for treating Huntington’s disease, a rare and inherited neurodegenerative disorder. This designation comes after early clinical trials indicated that AMT-130 may meaningfully slow the disease’s progression, offering hope where no current treatment modifies the condition. The Breakthrough Therapy status is designed to expedite the development and review process for treatments that show significant advantages over existing options. This opportunity is a critical step forward in addressing the unmet medical needs of Huntington’s disease patients. Encouraged by the promising trial results, uniQure is committed to further developing AMT-130 and working closely with the FDA to ensure patients can access a potential transformative therapy as quickly as possible.
Read Announcement- Drug:
- AMT-130
- Announced Date:
- April 17, 2025
- Estimated Event Date Range:
- April 1, 2025 - June 30, 2025
- Target Action Date:
- Q2 2025
- Indication:
- Huntington's Disease
Announcement
uniQure N.V. announced that Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025
AI Summary
uniQure N.V., a leading gene therapy company, announced that additional regulatory updates and guidance on its Biologics License Application submission are expected in the second quarter of 2025. This key update represents an important step as the company works closely with the FDA to prepare for the regulatory review of AMT-130, a gene therapy aimed at treating Huntington’s disease. The anticipated guidance will help outline the necessary requirements for the application, providing a clearer roadmap for the submission process. uniQure hopes that this update will speed up the overall review and bring them closer to offering an effective treatment option. The extra insights from the FDA are expected to support uniQure as it advances AMT-130 into the next stages of clinical and regulatory evaluation, ultimately benefiting patients affected by Huntington’s disease.
Read Announcement- Drug:
- AMT-130
- Announced Date:
- February 27, 2025
- Indication:
- Huntington's Disease
Announcement
uniQure N.V. highlighted recent progress across its business.
AI Summary
uniQure N.V. recently shared that the past year has been transformative for its business, highlighting significant clinical and operational progress. The company has advanced its pipeline of innovative gene therapies, including AMT-130 for Huntington's disease, AMT-260 for mesial temporal lobe epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-ALS. Patient enrollments are progressing well across these Phase I/II studies, with key data expected to help shape future development plans. In addition, uniQure has streamlined its operations through a company-wide restructuring and the sale of its Lexington manufacturing facility, actions that have reduced cash burn and strengthened its financial position. With a robust cash reserve that supports operations into the second half of 2027, the company is poised to continue advancing its promising therapies toward potential regulatory submissions in the near future.
Read Announcement- Drug:
- AMT-130
- Announced Date:
- July 9, 2024
- Indication:
- Huntington's Disease
Announcement
uniQure N.V announced updated interim data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington's disease.
AI Summary
uniQure N.V. announced updated interim data from its ongoing U.S. and European Phase I/II trials of AMT‑130 for Huntington’s disease. The data, collected over a 24‑month follow‑up period from 29 treated patients, showed promising results. Patients receiving a high dose of AMT‑130 experienced an 80% slower progression of the disease, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared to a matched external control group. Additionally, both dosage groups had reduced levels of the neurodegeneration biomarker, CSF neurofilament light protein (NfL), falling below their baseline levels, with the high‑dose outcomes particularly notable. These dose‑dependent results support a potential long‑term clinical benefit of AMT‑130. uniQure plans to continue monitoring patient outcomes and will discuss the data with the FDA for potential expedited development later this year.
Read Announcement- Drug:
- AMT-130
- Announced Date:
- June 3, 2024
- Indication:
- Huntington's Disease
Announcement
uniQure N.V announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy AMT-130 for the treatment of Huntington's disease.
AI Summary
uniQure N.V. announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational gene therapy, AMT-130, for treating Huntington’s disease. This designation, the first of its kind for a Huntington’s disease therapy, was based on promising 24-month interim Phase I/II clinical data announced in December 2023. The RMAT status highlights AMT-130’s potential to address a significant unmet medical need in patients with this debilitating disorder. Moreover, RMAT designation enables uniQure to work more closely with the FDA, potentially expediting product development and approval processes. This increased collaboration may lead to earlier access for patients suffering from Huntington’s disease, marking an important milestone in the search for effective treatments. The company plans to provide further clinical data updates later in 2024.
Read Announcement
AMT-162 FDA Regulatory Events
AMT-162 is a drug developed by uniQure for the following indication: For the Treatment SOD1-ALS.
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:
- AMT-162
- Announced Date:
- January 30, 2025
- Indication:
- For the Treatment SOD1-ALS
Announcement
uniQure N.V. announced that the Independent Data Monitoring Committee (IDMC) for EPISOD1, uniQure's Phase I/II clinical trial of AMT-162, an investigational gene therapy for amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, has met and reviewed 28-day safety data from the first study cohort.
AI Summary
uniQure N.V. announced that the Independent Data Monitoring Committee (IDMC) for its Phase I/II clinical trial EPISOD1 has reviewed 28-day safety data from the first dose cohort of AMT-162, an investigational gene therapy for ALS caused by SOD1 mutations. The IDMC found no significant safety concerns, which allowed the team to move forward with dose escalation and enroll patients for the second cohort. This initial safety check is a key milestone in the development of AMT-162. The therapy uses an AAVrh10-based vector to deliver a microRNA that helps reduce the mutated SOD1 protein responsible for toxic effects on motor neurons. uniQure is optimistic about the progress and looks forward to advancing patient enrollment and further assessing the treatment’s potential benefit in slowing or halting ALS progression.
Read Announcement- Drug:
- AMT-162
- Announced Date:
- January 30, 2025
- Estimated Event Date Range:
- January 1, 2025 - March 31, 2025
- Target Action Date:
- Q1 2025
- Indication:
- For the Treatment SOD1-ALS
Announcement
uniQure N.V. announced that Company Expects to Initiate Enrollment of Second Dose Cohort in the First Quarter of 2025 ~
AI Summary
uniQure N.V. has announced a new step in their Phase I/II clinical trial of the gene therapy candidate AMT-162 for SOD1-ALS. The company revealed that they plan to begin enrollment for the second dose cohort in the first quarter of 2025. This decision comes on the back of positive safety data reviewed by the Independent Data Monitoring Committee (IDMC) from the first dose cohort. The IDMC found no significant safety concerns, which supports the company's decision to move forward with dose escalation in the upcoming cohort.
This progress marks another important milestone in uniQure's efforts to develop a one-time, intrathecal gene therapy for patients with SOD1-ALS. The enrollment and dose-escalation process in the second cohort is expected to help further assess the safety and potential efficacy of AMT-162 in addressing this severe condition.
Read Announcement
AMT-191 FDA Regulatory Events
AMT-191 is a drug developed by uniQure for the following indication: For Fabry disease.
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:
- AMT-191
- Announced Date:
- September 23, 2024
- Indication:
- For Fabry disease
Announcement
uniQure N.V announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure's investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-center, open-label Phase I/IIa trial of AMT-191.
AI Summary
uniQure N.V. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational gene therapy, AMT-191, for the treatment of Fabry disease, a rare inherited disorder. This special status is meant to boost the development of new treatments for patients who currently have limited options due to the suboptimal effectiveness of existing therapies.
In addition, in August 2024, uniQure dosed the first patient in its U.S. multi-center, open-label Phase I/IIa clinical trial of AMT-191. The trial aims to test the safety, tolerability, and early signs of efficacy of this one-time, intravenously administered gene therapy. With the Orphan Drug Designation, the company will benefit from incentives, including tax credits and market exclusivity, as it works toward bringing new treatment options for Fabry disease to patients.
Read Announcement- Drug:
- AMT-191
- Announced Date:
- August 15, 2024
- Indication:
- For Fabry disease
Announcement
uniQure N.V. announced that the first patient has been dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease, a rare, inherited genetic disease.
AI Summary
uniQure N.V. has reached an important milestone by dosing the first patient in its Phase I/IIa clinical trial of AMT-191, a gene therapy designed for Fabry disease—a rare, inherited condition. This trial, conducted at multiple U.S. sites, will assess the treatment’s safety, tolerability, and early signs of effectiveness using two dose-escalating groups. The study aims to generate clinical proof-of-concept data by evaluating well-established endpoints in Fabry disease.
AMT-191 uses an AAV5-based delivery system similar to that used in HEMGENIX®, known for its safety and effectiveness, even in patients with preexisting antibodies. With this innovative approach, uniQure hopes to provide a potentially one-time treatment that could offer significant benefits over the current enzyme replacement therapies for patients with Fabry disease.
Read Announcement
AMT-260 FDA Regulatory Events
AMT-260 is a drug developed by uniQure for the following indication: For Refractory Mesial Temporal Lobe Epilepsy.
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:
- AMT-260
- Announced Date:
- May 29, 2025
- Indication:
- For Refractory Mesial Temporal Lobe Epilepsy
Announcement
uniQure N.V. announced the presentation of a clinical case study of the first participant dosed in the first cohort of its ongoing Phase I/IIa trial of an investigational gene therapy candidate, AMT-260, in patients with refractory MTLE.
AI Summary
uniQure N.V. presented encouraging early data from the first participant dosed in the ongoing Phase I/IIa trial of its gene therapy candidate, AMT-260, for refractory mesial temporal lobe epilepsy (MTLE). The participant, who experienced about seven seizures per month before treatment, reported only two seizures during five months of follow-up, with no seizures in the last 60 days as of the April cutoff. Notably, this represents a 92% reduction in seizure frequency, and no serious adverse events were observed. Dr. Firas Taha shared these results at the Epilepsy Therapies & Diagnostics Development Symposium. The findings boost hope that AMT-260, designed to suppress the GRIK2 gene linked to seizure activity, could offer a valuable and safer treatment alternative for individuals with drug-resistant MTLE. Further follow-up and additional data from other participants are needed to confirm these early positive signals.
Read Announcement
