1800 244 735

FDA: further trial needed for Huntexil approval in HD

NeuroSearch, the developer of experimental Huntington’s disease drug Huntexil, has reported on their meeting with the FDA. The FDA requires that another trial be conducted before Huntexil could be approved in the US.

What is Huntexil?

As previously discussed on HDBuzz, Huntexil is an experimental drug, specifically developed to help with some of the movement problems associated with Huntington’s disease. Also known as pridopidine and ACR16, Huntexil is made by a Danish company, NeuroSearch.

What trials have been done with Huntexil?

NeuroSearch sponsored two trials of Huntexil in HD patients. The first involved 437 patients in the European Huntington Disease network, called the ‘MermaiHD’ trial. The second, with the Huntington Study Group in North America, was called the ‘HART’ study and included 227 patients.

The MermaiHD trial was frustratingly inconclusive. While some improvement was seen in patients' movement symptoms, the findings weren’t robust enough to call the trial a definite success. Something very similar happened with the HART study – improvements in movement symptoms were seen, but they weren’t strong enough to be conclusive.

NeuroSearch pooled the results of the MermaiHD and HART trials together, in what is known as a ‘meta-analysis’. This allows scientists look at the results from multiple studies together. That analysis did show a robust improvement in the movement symptoms in HD patients. But it wasn’t clear whether that would be enough to get the drug approved.

In general, the regulatory agencies that approve drugs for patient use don’t allow this kind of ‘meta-analysis’ to be used as evidence for a drug being effective. Usually, the results of a single, planned trial are presented and considered as evidence. Still, NeuroSearch said that they would present the results of their meta-analysis to the FDA, and argue that it was sufficient proof that Huntexil worked.

What’s just happened?

On March 23rd, NeuroSearch announced that they’d had a meeting with the FDA called an “end of phase II meeting”. At this meeting, the FDA told NeuroSearch that they would need “additional clinical evidence” to apply for approval for Huntexil in the USA. That means that the FDA would require the results of a further clinical trial before Huntexil could be approved.

Now what?

NeuroSearch has also applied for approval for Huntexil from the European Medicines Agency (EMA). They expect to hear from the EMA sometime by June 2011 whether the meta-analysis they conducted will be enough to get Huntexil approved for use in Europe.

As for the USA, NeuroSearch will have to run another trial to get approval for Huntexil. CEO, Patrik Dahlen, said in a press release that their plan for new trials “is currently being developed”. Their decision will likely be influenced by whether Huntexil is approved in Europe or not.

Latest Research Articles

Understanding expansions at the single cell level

Published date: 12 March, 2024

In two recent studies, researchers looked at how different parts of the brain are affected by CAG expansions in Huntington’s disease (HD) at the level of individual brain cells. The scientists looked at post-mortem brains from people with and without HD to track molecular changes in different brain regions called the cortex and striatum. These ... Read more

Huntington's Disease Therapeutics Conference 2024 – Day 3

Published date: 7 March, 2024

HDBuzz is back for the last day of the CHDI HD Therapeutics Conference: Thursday February 29th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. From genes to medicines The morning session will focus on how human genetics is driving the development of therapeutics. “Genetic modifiers” are genes ... Read more

Huntington's Disease Therapeutics Conference 2024 – Day 2

Published date: 6 March, 2024

HDBuzz is back for Day 2 of the CHDI HD Therapeutics Conference: Wednesday February 28th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. It’s a brain disease This morning’s session is titled “It’s a brain disease” and will feature talks about BRAINSSSS! HD scientists are a bit ... Read more

Huntington's Disease Therapeutics Conference 2024 – Day 1

Published date: 5 March, 2024

The HDBuzz team recently convened in Palm Springs, California, along with hundreds of other scientists from all over the globe, for the 19th Annual HD Therapeutics Conference, hosted by the HD research foundation CHDI. From Tuesday February 27th through Thursday February 29th, we live-tweeted dozens of scientific talks by world experts in Huntington’s disease research, ... Read more

CRISPR-based drugs: one giant leap for mankind

Published date: 14 February, 2024

You’ve likely heard of CRISPR. By now, you also may have heard that CRISPR has been used to produce a revolutionary new treatment for Sickle Cell Disease. Just 4 years after the Nobel Prize for the discovery of CRISPR was awarded, we have an approved treatment using this technology. This may have you wondering if ... Read more

Making babies: having a family, the HD way

Published date: 11 January, 2024

For people at risk of Huntington’s disease, having a baby who might inherit HD can make decisions around planning a family extremely difficult. This article explains the options available, and how modern reproductive science can make a difference right now to families touched by HD. Content warning This article describes issues of fertility, tough choices, ... Read more