AMT-130 Hits Another Milestone in Quest to Treat Huntington's Disease


uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.

Just days after uniQure announced plans to advance the development of AMT-130, an investigational gene therapy for the treatment of Huntington’s disease (HD), the company was granted Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA).

Last week, uniQure stated its intentions to file an investigational new drug (IND) application with FDA in the second half of 2018. The first human clinical trial with the drug is expected to begin soon after the FDA’s projected acceptance. The drug was granted orphan drug designation by the FDA in October.

"The granting of orphan drug designation in Europe represents another important milestone for our AMT-130 program,” said Matthew Kapusta, chief executive officer of uniQure in a press release. “Huntington’s disease affects approximately 70,000 people in the U.S. and Europe1, making this one of the largest clinical unmet needs in the rare disease field. We expect to file an Investigational New Drug application later this year and be the first AAV-gene therapy to enter clinical development for Huntington’s disease.”

In HD, CAG trinucleotide expands in exon 1 of a multifunctional gene coding for the huntingtin protein. The genetic neurodegenerative disorder can lead to the loss of muscle coordination, behavioral abnormalities and cognitive decline. Over time, it can lead to complete physical and mental deterioration. AMT-130 is a huntingtin-lowering approach that would be mediated via the viral delivery of adeno-associated virus (AAV).

Using AAV vectors, the drug is being developed to deliver micro-RNAs directly into the brain, and inhibiting the production of the mutant protein.

Current treatment options for HD patients are limited to alleviation from symptoms, but they do not delay the onset or slow progression. It is widely believed that AMT-130 has the potential to completely stop the disease.

“We ended 2017 with significant momentum across all of our programs and the cash to fund operations into 2020,” stated Kapusta in a previous statement. “Now that we have established robust, commercial-scale manufacturing in our Lexington facility, we are highly focused on the near-term initiation of our pivotal study of AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in Huntington’s disease.”

For the latest on AMT-130 and the development of the first potential therapy for Huntington’s disease, follow Rare Disease Report on Facebook and Twitter.

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