On September 23rd, 2024, the pharmaceutical company Biohaven announced positive topline results from their study of the drug Troriluzole to treat SCAs 1,2,3,6,7,8 and 10.
Those with SCA who were treated with Troriluzole showed a 50 – 70% slowing of disease progression, compared to those with SCA who were untreated over a 3-year study period. This represents a 1.5 – 2.2 years delay in disease progression over the 3 years. The study compared results of people on the Biohaven troriluzole trial (including people with SCA1,2,3,6,7,8 and 10) with data from people on natural history studies, such as the European study EuroSCA. Many people from the UK took part in EuroSCA at the London Ataxia Centre; we are grateful to these study participants for their vital contribution to research that can lead to the development of treatments.
Following these results, Biohaven plans to submit a new drug application for Troriluzole for all types of SCAs to the US Food and Drug Administration (FDA) in the last quarter of 2024. As we reported previously, Troriluzole is also being evaluated by the European Medicines Agency (EMA) to treat SCA3, and we are awaiting information on the outcome of this. This new data should provide additional evidence on the benefit of the drug. Ataxia UK will be encouraging Biohaven to seek approval in the UK and we will share any information as soon as we have it.
The results of this study will be presented at the International Congress for Ataxia Research (ICAR) in London in November.
Read the press release from Biohaven on their website here.