Accelerated evaluation of drug

The European Medicines Agency has acknowledged the huntingtin lowering drug, RG6042 (previously known as IONIS-HTTRx), as a possible therapy that may significantly improve patients’ quality of life and granted Priority Medicine designation.


In other words, with the “PRIME” (PRIority MEdicine) designation, the European Medicines Agency will assist the pharmaceutical company in making the drug available as quickly as possible.

Astri Arnesen, President of EHA.

– The Agency will provide early and proactive support to Roche to accelerate the evaluation of RG6042 with the hope that it will help patients to benefit as early as possible, says May Lise Nguyen, Patient Partnership Director at Roche.

This can save the Huntington community for valuable time.

– This is such good news! I am so grateful that EMA recognize the great need for treatment in Huntington’s disease, says Astri Arnesen, President of the European Huntington Association.

Positive results

RG6042 have been tested in 46 participants with positive results. The drug showed both to be safe, well-tolerated and to actually lower the level of the toxic Huntingtin protein in the brain.

This Huntingtin lowering has further shown to be correlated with improved symptoms in people with Huntington.


The drug developing process: 
In the pre-clinical phase, a treatment is tested in animals. If it’s safe, it’s further tested in a clinical trial, where humans participate. Here the drug is tested in different phases: safety first, then efficacy. The last step is to get the drug approved and available on the market.

Next step

The next step is to confirm the drug’s safety and efficacy in a larger group of people.

If the upcoming study shows the same positive results, the European Medicines Agency will evaluate the drug as quickly as possible and make it available for patients in Europe.

– Now, I am only waiting for the trial to start recruiting participants and get started. We still don’t know when it’s due but I know Roche is doing their best to make it happen as soon as possible, Arnesen says.

More knowledge over time

The only thing family members impatiently waiting can do is to give it time. It’s only over time researchers really know whether the drug is safe, what it does to the body and what the body does to the drug.

– Pre-approval access programs, compassionate use and “right to try” requests for RG6042 cannot yet be supported because more evidence is needed regarding efficacy and safety to meet the requirements of regulatory authorities, Nguyen states.

RG6042 is therefore only accessible through participation in a clinical trial at this time.

You can find Roche’s full press release here.