Omaveloxolone, the first drug for Friedrich's ataxia.

This paper below tells more about how perhaps our ATH434 could work with this new drug as it is an iron chelator and iron overload in FA is a big problem. This is the abstract but you need to go to the full text.

Review

Trends Mol Med

. 2024 Feb;30(2):117-125.

doi: 10.1016/j.molmed.2023.12.002. Epub 2024 Jan 24.

Omaveloxolone: a groundbreaking milestone as the first FDA-approved drug for Friedreich ataxia

Federica Pilotto ¹, Deepika M Chellapandi ¹, Hélène Puccio ²

Affiliations expand

• PMID: 38272714

DOI: 10.1016/j.molmed.2023.12.002

Abstract

Friedreich ataxia (FA) is an inherited autosomal recessive neurodegenerative disease (NDD) characterized primarily by progressive sensory and spinocerebellar ataxia associated with hypertrophic cardiomyopathy. FA is due to an intronic GAA repeat expansion within the frataxin gene (FXN) leading to reduced levels of frataxin (FXN) which causes mitochondrial dysfunction, production of reactive oxygen species (ROS), and altered iron metabolism. To date there is no resolutive cure for FA; however, the FDA has recently approved omaveloxolone - a potent activator of nuclear factor erythroid 2-related factor 2 (NRF2) - as the first treatment for FA. We discuss herein the urgency to find a resolutive cure for NDDs that will most probably be achieved via combinatorial therapy targeting multiple disease pathways, and how omavaloxolone serves as an example for future treatments.

Keywords: Friedreich’s ataxia; NRF2 pa