uniQure, a prominent gene therapy company dedicated to advancing transformative treatments for patients with severe medical conditions, has announced a significant milestone. The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation for uniQure's investigational gene therapy, AMT-130, designed for the treatment of Huntington’s disease.
The RMAT designation was awarded based on the potential of AMT-130 to address the critical unmet medical needs of individuals affected by Huntington’s disease. This recognition follows the FDA's review of interim Phase I/II clinical data for AMT-130, which was disclosed in December 2023. The analysis compared the 24-month clinical data to a non-concurrent criteria-matched natural history cohort.
Matt Kapusta, the chief executive officer of uniQure, expressed excitement about receiving the first-ever RMAT designation for an investigational therapy for Huntington’s disease. He highlighted the significance of this achievement in supporting the potential of AMT-130 to address the substantial medical needs of patients grappling with this devastating disease.
Walid Abi-Saab, the chief medical officer of uniQure, emphasized the promising start to their interactions with the FDA facilitated by the RMAT designation. He underscored the importance of increased collaboration with the FDA to expedite development, potentially enabling earlier access to life-saving treatments for patients with critical medical conditions. The uniQure team looks forward to presenting updated interim data from their ongoing Phase I/II studies later in the year.
The RMAT designation, established as part of the 21st Century Cures Act, aims to streamline the development and review processes of regenerative medicine therapies. To qualify for RMAT designation, a regenerative medicine therapy must demonstrate the potential to treat, modify, reverse, or cure a serious condition with preliminary clinical evidence indicating its ability to address unmet medical needs. Sponsor companies, like uniQure, benefit from early, close, and frequent interactions with the FDA, paving the way for accelerated development and potential approval pathways.
uniQure plans to provide updated interim data from the ongoing Phase I/II studies of AMT-130 in the middle of 2024. This update will include up to three years of follow-up on 29 treated patients, with 21 patients having been monitored for at least two years. The Phase I/II clinical program of AMT-130 in the U.S. involves assessing safety, tolerability, and efficacy signals in patients with early manifest Huntington’s disease, with a focus on two dose cohorts.
The European Phase Ib/II study of AMT-130 aims to establish safety, proof of concept, and the optimal dose to advance into Phase III development. Patient dosing is ongoing in a third cohort to further evaluate both doses of AMT-130 in combination with perioperative immunosuppression. AMT-130 represents uniQure's inaugural clinical program targeting the central nervous system, leveraging its proprietary miQURE® platform.
Huntington’s disease, a rare inherited neurodegenerative disorder, leads to motor symptoms, behavioral abnormalities, and cognitive decline, resulting in progressive physical and mental deterioration. Despite its known etiology, there are currently no approved therapies to delay the onset or slow the disease’s progression. uniQure remains committed to revolutionizing medicine by delivering innovative cures that transform lives, with a focus on developing gene therapies for severe diseases like Huntington’s disease.
