The Neurology Year in Review Session, which will cover amyotrophic lateral sclerosis (ALS), will cover an entire landscape over the past year that’s been pretty exciting in ALS. In September 2022, we had two new therapies that were approved by the FDA. One was edaravone, taken orally, and the other was sodium phenylbutyrate and taurursodiol, also known as relyvrio. In the following year, in 2023, we had an unprecedented, accelerated approval for a gene-targeted therapy called tofersen that was targeting SOD1-ALS...
The Neurology Year in Review Session, which will cover amyotrophic lateral sclerosis (ALS), will cover an entire landscape over the past year that’s been pretty exciting in ALS. In September 2022, we had two new therapies that were approved by the FDA. One was edaravone, taken orally, and the other was sodium phenylbutyrate and taurursodiol, also known as relyvrio. In the following year, in 2023, we had an unprecedented, accelerated approval for a gene-targeted therapy called tofersen that was targeting SOD1-ALS. This is the first ever gene therapy approved for ALS, targeting a specific form which affects about 1 to 2% of ALS.
What’s interesting is in the last few months, we’ve had a lot of news. One is that tofersen got a positive opinion at CHMP in Europe, so it looks like they’ll get a conditional approval in Europe. Also, there was a confirmatory Phase III of relyvrio conducted in mainly in Europe (but it was a global study), that did not hit its primary endpoint and is being presented at the American Academy of Neurology. This led to the manufacturers leading a press release in the last couple of weeks, stating their intention to withdraw the medication from the market in both the US and Canada. Despite the disappointing results, I think one of the big takeaways from this is that the regulatory process worked. It’s already been conducted in other disease areas, but for ALS, this was an example of getting something that had some degree of benefit – it was unclear and a confirmatory study was required- but the FDA provided regulatory flexibility to allow it to come to market so that people who are dying with ALS could actually have access and try it, while we’re trying to confirm the results from the Phase II study. Having the product evaluated in a confirmatory Phase III study, and then making decisions to whether to keep it on the market or not, I think was something that showed that the regulatory flexibility and showed that the process worked for our community.
