We would like to share an important update released today by uniQure regarding AMT-130 after their recent meeting with the FDA, the regulatory agency in the United States.
The purpose of the meeting was to discuss plans for submitting a Biologics License Application (BLA), which is a formal request to the FDA for permission to market a biological product, in this case AMT-130. It is a process similar to what in Europe would be a Marketing Authorisation Application (MAA), which is submitted to the European Medicines Agency (EMA).
The FDA expressed that the current data from the ongoing Phase I/II study of AMT-130, compared to an external control group, may no longer be sufficient on its own to support a BLA submission. This represents a major change from the previous guidance uniQure had received from the agency.
“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway,”
– Matt Kapusta, CEO at uniQure
As a result, the initially expected timeline for submitting the approval application has changed and now remains uncertain. uniQure is expecting to receive more information from the FDA within the next 30 days.
It is important to note that this update is merely regulatory and does not change the data that was shared a few weeks ago, which we explained in a previous article. The clinical results remain encouraging and continue to suggest potential benefits for people living with Huntington’s disease. What has changed is the FDA’s position on how and what the data should be evaluated for approval purposes in the U.S..
The current situation puts in evidence the kind of challenges that can arise during the development and approval process of innovative therapies, especially in rare diseases. We still don’t know what the next steps will be and what data will be required for the submission, but uniQure has expressed both their disappointment and commitment to finding the best path forward.
“This news is unexpected, and we are truly disappointed for people living with HD, who have no treatment options for this devastating disease. We strongly believe AMT-130 can provide substantial benefit to patients, and remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.”
– Matt Kapusta, CEO at uniQure
In parallel, the company plans to continue conversations with the regulatory authorities in Europe and the United Kingdom, which follow their own independent review processes.
We understand that this news brings uncertainty, but we want to highlight that the science and the results remain the same, and that efforts to advance AMT-130 still continue.
We will keep the community informed as new information becomes available.
You can read both the community letter here and the press release here.
