The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to uniQure’s AMT-130 for treating Huntington’s disease, a rare neurodegenerative condition.
This designation for the therapy adds to a list of the agency鈥檚 recognitions, including regenerative medicine advanced therapy (RMAT), fast track and orphan drug designations.
Clinical data from the ongoing Phase I/II studies of the therapy support this latest designation.
Interim data presented by the company last July demonstrated a dose-dependent slowing of progression of the disease in individuals treated with the therapy, against a propensity-weighted natural history. Till now, 45 subjects received the treatment.
uniQure chief medical officer Walid Abi-Saab said: 鈥淩eceiving breakthrough therapy designation underscores both the urgent need for effective treatments for Huntington鈥檚 disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression.
鈥淚t鈥檚 a powerful recognition of the promise of AMT-130 and the important progress we鈥檝e made. We deeply value the FDA鈥檚 continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington鈥檚 disease patient community as quickly as possible.鈥�
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By GlobalDataThe breakthrough therapy status is granted to accelerate investigational therapies development for serious conditions when preliminary clinical evidence suggests a substantial improvement over existing treatments.
uniQure noted that Huntington’s disease, which results in chorea, cognitive decline, and behavioural abnormalities, affects approximately 70,000 people in Europe and the US, with many more at risk.
The company鈥檚 gene therapy for haemophilia B has paved the way for new treatment approaches in genomic medicine.
uniQure is currently progressing with its pipeline of gene therapies aimed at treating Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, amyotrophic lateral sclerosis (ALS), and other severe conditions.
