Developer now seeking approval of Huntington’s gene therapy in US, UK
1-time treatment would be administered directly to brain
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uniQure has submitted an application to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of AMT-130, the Netherlands-based company’s gene therapy candidate for Huntington’s disease.
A similar request for marketing authorization in the U.K. has been made to the nation’s Medicines and Healthcare products Regulatory Agency (MHRA), uniQure announced.
The one-time gene therapy, administered directly into the brain via a surgical procedure, is not a cure for the genetic disorder, but is designed to slow Huntington’s progression. According to uniQure, it aims to silence the gene that drives the disease, which is marked by symptoms such as movement problems, cognitive impairment, and behavioral issues.
“The submission of licensing applications for [AMT-130] represents an important milestone for the Huntington’s disease community,” Matt Kapusta, uniQure’s CEO, said in a company press release providing updates on the treatment’s development.
“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,” Kapusta added. “We look forward to working with both agencies as these applications progress.”
Huntington’s is caused by genetic mutations that lead to the production of an abnormal version of the huntingtin protein, which is thought to drive the disease. AMT-130, also known as ifezuntirgene inilparvovec, aims to slow the progression of Huntington’s by shutting down the production of huntingtin protein in the brain.
AMT-130 slowed disease progression by 75% in 2 trials
The FDA’s accelerated approval program allows an experimental therapy to be brought to market based on early evidence that it will likely benefit patients. If AMT-130 is granted this approval, uniQure will be required to conduct additional clinical testing to demonstrate that the treatment benefits patients, with long-term authorization contingent on the results. UniQure said it’s already working on plans for a confirmatory trial.
The company is asking the FDA to grant this approval to AMT-130 based on three-year data analyses from two Phase 1/2 clinical trials — one in the U.S. (NCT04120493) and one in Europe (NCT05243017). These data showed that a high dose of AMT-130 slowed disease progression by 75% compared with an external group of untreated patients in natural history studies. uniQure said it will present four-year data from these studies in the next few months.
The FDA had previously requested that an additional trial be run before uniQure submitted an application for AMT-130. But then this summer, following a meeting with uniQure, the FDA dropped that request and agreed that data from these trials could support an application seeking approval.
Usually, it takes about 10 months from the time the FDA accepts an application to the time it issues a decision. uniQure has asked the FDA to grant priority review to its application for AMT-130, which would shorten the review time to about six months.
The FDA has granted AMT-130 several designations aimed at speeding the development of therapies that may fill unmet medical needs, including regenerative medicine advanced therapy status and fast track and breakthrough therapy designations.
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