The recent report that pharmaceutical company uniQure’s data from its Phase I/II studies provide insufficient evidence for the approval of its investigational gene therapy drug for Huntington’s disease (HD) are a major blow for patients. 

Huntington’s disease is a rare genetic disorder that can affect around 5-10 people per 100,000, and which is more dominant in those of European descent. Currently, there is no cure for the disease, which is highly impactful and develops over time through a range of symptoms resulting in loss of bodily functions and eventually death.  

As such as impactful disease, last year’s positive results from uniQure were an exciting development for an area of neuroscience that has had little progress in terms of actual cures. So far, treatments have been focused on managing symptoms of the disease for better quality of life for patients.  

uniQure’s results showed for the first time a therapy that displayed continuing and statistically significant slowing of the disease. Other exciting developments for the gene therapy included the fact that it was well tolerated by patients and that one dose was expected to last a person’s whole life.  

There was a notable air of excitement by industry and healthcare surrounding the data. I myself spoke in a DDW podcast this year about expected advancements in neuroscience and uniQure’s position as a leader in this space.  

Whilst the data is still positive, the FDA’s opinion that the company’s phase I/II results are not sufficient enough evidence to support a marketing application for AMT-130 has resulted in a major setback for uniQure. The FDA’s decision meant that investors in uniQure launched a class action lawsuit to recover losses, stating that uniQure made false statements surrounding its clinical studies and alleging that uniQure downplayed the likelihood of having to delay its Biologics License Application (BLA) to perform additional studies.  

Somewhat of an egg-on-face moment for the gene therapy company then that really highlights the importance of investor-client transparency.  

Indeed, a recent article in DDW, written by the law firm Hogan Lovells, explored this very topic, with authors writing that: “ Cutting-edge research and products also need to be fairly presented so as not to oversell current functionality or mislead investors as to how much progress has actually been made. While a company may be confident that its product will revolutionise the industry, it cannot imply that it currently has functions it does not actually possess.”  

The case for uniQure is yet to be determined and the company is actively working with the FDA towards Phase III development and potential study design approaches. As we know though, this is an industry backed by both science and money, and the recent fall in uniQure’s share prices could prove extremely detrimental to the company’s development plans moving forward.  

Fortunately, the company is buoyed by very promising data for a product that could prove extremely impactful in a clinical setting with great need. As such, it may just survive this set back. Here’s hoping, as the biggest loser in all of this won’t be the investors, it will be the patients.  

From DDW Volume 27 – Issue 2, Spring 2026 – Read the digital issue here