Drug company Prilenia has announced today, Tuesday 12 March, that it is applying for market authorisation from the European Medicines Agency (EMA) for the drug Pridopidine for the treatment of Huntington’s disease.

Marketing authorisation is the process of reviewing and assessing the evidence to support a medicinal product in relation to its marketing, finalised by granting of a licence to be sold if approved.

This is the first time that a drug developed to treat Huntington’s disease has reached this stage. Whilst a notable milestone, the Huntington’s community should remain mindful that this is the beginning of a potentially lengthy process with no guarantee of a positive outcome.

Pridopidine was the drug used as part of the PROOF-HD trial, which included a research site in Aberdeen (https://www.abdn.ac.uk/news/14828/). 

In April 2023 Prilenia announced the findings from the PROOF-HD study, which unfortunately did not meet its primary or secondary endpoints. Nevertheless the manufacturer remains hopeful that the treatment can still potentially provide benefit to patients.

Key points to keep in mind:

• Pridopidine is NOT intended to be a “cure” for Huntington’s disease. The manufacturer believes that the treatment shows potential for a slowing of disease progression.
• The review of the application to be licenced by the EMA takes time, potentially up to a year from application. EMA can reject the application.
• EMA authorisation covers the EU, Norway, Iceland and Lichtenstein only. If the EMA agrees to licence the treatment it will still take time before the medicine is potentially available across these jurisdictions. Prilenia will need to go to the relevant authorities in each country and negotiate market access and pricing. One of the key questions at that stage would be affordability compared to patient benefit.
• Prilenia will need additional permission from authorities in the UK to market the drug here, which it would presumably seek to do if EMA approval is secured.

“Whilst we of course welcome news that a drug developed to treat Huntington’s disease has reached this stage for the first time, and the hope that this provides for the Huntington’s community, we remain mindful of the significant hurdles that lie ahead before patients could potentially benefit from this treatment,” said Scottish Huntington’s Association Chief Executive Alistair Haw.

Read Prilenia’s letter to the Huntington’s community here.