LEXINGTON, Mass. and AMSTERDAM, Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the submission of a Biologics License Application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington’s disease. The Company also announced that its Marketing Authorisation Application (MAA) for ifezuntirgene inilparvovec has been submitted to the United Kingdom’s (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA).
“The submission of licensing applications for ifezuntirgene inilparvovec represents an important milestone for the Huntington’s disease community,” said Matt Kapusta, chief executive officer at uniQure. “We are grateful to the FDA for its leadership in advancing regulatory science to meet the urgency of this disease, and to the MHRA for its commitment to advancing rare disease treatments in the U.K. We look forward to working with both agencies as these applications progress.”
The Company has requested priority review for the BLA. If granted, priority review would shorten the FDA review cycle to six months following the FDA’s 60-day BLA filing review period.
The BLA and MAA are supported by the previously announced three-year data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database. The Company intends to present a four-year data analysis from the ongoing Phase I/II clinical studies before the end of the current third quarter.
Ifezuntirgene inilparvovec is the first investigational therapy for Huntington’s disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Ifezuntirgene inilparvovec also holds Fast Track designation from the FDA.
About Ifezuntirgene Inilparvovec (AMT-130)
Ifezuntirgene inilparvovec is a novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes a proprietary, gene-silencing miQURE® platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment. Treated patients receive a single administration through targeted, MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).
About the Phase I/II Clinical Program of Ifezuntirgene Inilparvovec
uniQure is conducting two multi-center Phase I/II clinical studies evaluating the safety, tolerability, and efficacy of ifezuntirgene inilparvovec for the treatment of Huntington’s disease.
The U.S. randomized study enrolled 26 patients who received either a single administration of ifezuntirgene inilparvovec (n=6 low dose; n=10 high dose) or a sham procedure (n=10); four control patients subsequently crossed over to treatment after approximately 12 months. The European open-label study enrolled 13 patients (n=6 low dose; n=7 high dose). A third cohort of 12 patients explored both doses in combination with immunosuppression, and a fourth cohort of six U.S. patients is evaluating the high dose in patients with lower striatal volumes compared to those of patietns enrolled in previous cohorts.
Additional details are available on www.clinicaltrials.gov (
About Huntington’s Disease
Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Approximately 75,000 people have Huntington’s disease in the U.S.1, EU2, and the UK3, with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington’s disease, there are currently no approved therapies to delay the onset or to slow the disease’s progression.
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, and other severe diseases. For more information please visit www.uniQure.com.
References
1 Yohrling G, et al. Neurology 2020;94(15 Suppl):954.
2 Medina A, et al. Mov Disord 2022;37(12):2327–2335
3 Furby H, et al. Eur J Neurol 2022;29(8):2249–2257.
| uniQure Contacts: | |
| FOR INVESTORS: | FOR MEDIA: |
| Chiara Russo | Tom Malone |
| Direct: 781-491-4371 | Direct: 339-970-7558 |
| Mobile: 617-306-9137 | Mobile:339-223-8541 |
| [email protected] | |