uniQure sets course to US filing for Huntington gene therapy

uniQure has agreed with the FDA that it can proceed with a marketing application for Huntington's disease gene therapy AMT-130, using data that it already has on hand, rather than running another trial.

The FDA refused to accept uniQure's filing for AMT-130 last November, and earlier this year the Dutch biotech said it had been unable to persuade the US regulator that data from its phase 1/2 trial of the gene therapy, which compared it to an external control, would be sufficient.

Fast forward to today, and uniQure has announced the FDA will allow three-year data from the trial to serve as "the primary basis of a biologics license application for accelerated approval," setting up a filing in the third quarter of this year – and a potential approval in 2027.

Huntington's, which affects an estimated 30,000 to 41,000 people in the US and is classed as a rare disease, causes neurons in the brain to decay over time and leads to physical deterioration, as well as cognitive and mental health problems. AMT-130 is designed to switch off a mutation in the HTT gene that drives the disease using RNA interference, preventing the production of a toxic form of the huntingtin protein that attacks nerve cells.

In uniQure's phase 1/2 trial, the therapy was associated with a statistically significant 75% slowing of disease progression three years after a one-shot treatment, compared to historical control data.

"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA's genuine commitment to addressing the unmet need of Americans living with Huntington's disease," said Matt Kapusta, uniQure's chief executive.

"The consistency and strength of the clinical data generated to date give[s] us great confidence in the product's potential to make a meaningful difference for patients."

It's worth noting that uniQure still has to wait for the formal minutes of its last meeting with the FDA before it can proceed with its filing plans.

Meanwhile, the company's beleaguered share price – which was trading above $70 before the FDA's refusal-to-file (RTF) decision, but was at $26.99 at yesterday's close – had shot up 75% to more than $47 at the time of writing.

There is intense interest in the progress with AMT-130 as, at the moment, there are no disease-modifying therapies that can slow disease progression approved for Huntington's, with all current drugs tackling symptoms, such as the involuntary movements (chorea) that characterise the disorder.