SKYCLARYS™ (omaveloxolone) Approval by Health Canada Ushers in a New Era for Friedreich’s Ataxia Treatment in Canada

TORONTO, March 17, 2025 /CNW/ - Biogen Canada Inc. is pleased to announce that Health Canada has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients 16 years of age and older.2 This approval – granted under Health Canada’s Priority Review process – marks an important milestone making SKYCLARYS the only treatment in Canada to specifically target the underlying mechanisms of this rare, progressive neurodegenerative disease.

P732: Identification of pathogenic expansions within FXN in individuals with suspected Friedreich ataxia diagnoses

P732: Identification of pathogenic expansions within FXN in individuals with suspected Friedreich ataxia diagnoses, Bohm, Kaitlynne et al., Genetics in Medicine Open, Volume 3, 103101 doi:10.1016/j.gimo.2025.103101 

Our PCR and NGS-based test designed to detect pathogenic GAA repeat expansions and sequence variants within the FXN gene was successful in providing 18 positive test results to individuals known to have, or suspected of having, Friedreich Ataxia. The FA Identified sponsored testing program has presented a unique opportunity to understand the milieu of clinical characteristics of individuals with a positive FA diagnosis, and the health care providers seeking such testing for their patients.

P356: Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations

P356: Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations, Clayton, Russell et al., Genetics in Medicine Open, Volume 3, 102321. doi: 10.1016/j.gimo.2025.102321 

Modeling and simulations using PK and PD data from short term studies can be used to predict a potential long term therapeutic dose. In the majority of patients with FRDA, daily administration of 50 mg nomlabofusp is predicted to result in skin frataxin concentrations that are ≥ 50% of concentrations observed in healthy controls.

P289: Disease characteristics and tissue frataxin concentrations in adults with Friedreich’s ataxia participating in nomlabofusp interventional studies

Russell Clayton, Mohamed Hamdani, Noreen Scherer,, P289: Disease characteristics and tissue frataxin concentrations in adults with Friedreich’s ataxia participating in nomlabofusp interventional studies, Genetics in Medicine Open, Volume 3, Supplement 2, 2025, 102254, ISSN 2949-7744, doi10.1016/j.gimo.2025.102254. 

Tissue frataxin concentration data from these studies are consistent with prior studies demonstrating that lower frataxin concentrations are associated with earlier onset of disease. The range of characteristics observed in the population of patients with FRDA participating in the nomlabofusp interventional studies is representative of the general FRDA adult population. Buccal and skin cell frataxin levels correlate with each other.