Skyclarys and Oxidative Stress

[powerpadman]

Skyclarys was approved in Feb. 2023 for the treatment of Friedreich ataxia. For those who may not be aware, FA is an autosomal-recessive genetic disease which has some similarities to ALS.

I personally know some FA patients who have been taking Skyclarys and are benefiting from the drug which activates the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway that is involved in the cellular response to oxidative stress.

However, Skyclarys is not indicated for ALS, so any potential prescription would be off label and very expensive at $370,000 annually.

I was wondering if it could be worth pursuing Skyclarys as an option for ALS in the future? Potentially through an Institutional Review Board?

The two white papers below are helpful. The first outlines the potential need/benefit of an NRF2 activator for ALS and the second is an article with links to the Skyclarys clinical trial results.

Impaired antioxidant KEAP1-NRF2 system in amyotrophic lateral sclerosis: NRF2 activation as a potential therapeutic strategy - Molecular Neurodegeneration
Omaveloxolone Sustains Benefit Effect in Friedreich Ataxia at 3 Years

For those who may be more connected to the science, trials, and future state of ALS, I would be curious in hearing your opinions on this. Is this worth pursuing? Or are we barking up the wrong tree? Thank you.

 

[Nikki J]

Biogen would absolutely be pursuing this if they thought it had promise for als. A second indication for a more common disease on an existing drug is a great thing for a drug company. I haven’t heard any indication they are thinking of this but if they are we will see a trial probably sooner than later.