Biogen Disappointed by INESSS Recommendation on SKYCLARYS™ and its Impact on Quebec Patients with Friedreich Ataxia

TORONTO, June 5, 2025 /CNW/ - Biogen Canada Inc. is disappointed by INESSS's recommendation against listing SKYCLARYS™ (omaveloxolone), despite its therapeutic potential for individuals living with Friedreich ataxia (FA) — a community in urgent need of treatment options.

Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies

Fogel, B.L., Klopstock, T., Lynch, D.R., Maltecca, F., Verma, M., Minassian, B.A., Platt, F.M., Gonçalves, D.F., Puccio, H., Roos, A. and Synofzik, M. (2025), Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies. Ann Neurol. doi:10.1002/ana.27271 

 Autosomal recessive cerebellar ataxias (ARCAs) represent over 200 clinically heterogeneous genetic conditions involving degeneration of the cerebellum and associated tracts with resultant impairment of balance and coordination. Advancements in genomic testing have enabled rapid identification of the majority of known recessive disorders, shifting research focus to the development of targeted mechanistic treatments addressing underlying physiological pathways. Molecular classification allows recognition of cellular, biochemical, and genetic targets for high-effect precision therapy development. ARCAs represent a significant global health burden, requiring establishment of a robust pathway for novel therapeutic discovery through modification of mechanisms of disease pathogenesis and subsequent clinical trial development.

Health-related quality of life and productivity burden for non-professional caregivers of adults with rare diseases: a real-world study

Johnson, B., Gibson, G., Baskerville, D. et al. Health-related quality of life and productivity burden for non-professional caregivers of adults with rare diseases: a real-world study. Orphanet J Rare Dis 20, 282 (2025). doi:10.1186/s13023-025-03796-z 

The substantial burden of providing non-professional caregiving to adults with rare diseases is associated with multiple factors. Interventions improving care recipient HRQoL could enhance caregiver HRQoL and productivity.

Biogen Initiates Phase 3 Pediatric Study Of Omaveloxolone For The Treatment Of Friedreich Ataxia

CAMBRIDGE, Mass., June 18, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the initiation of dosing in the BRAVE study, a global Phase 3 clinical trial. The BRAVE study will evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) between the ages of 2 to < 16 . Both non-ambulatory and ambulatory participants may qualify for the study. Participants will be randomized 2:1 to receive omaveloxolone or placebo once a day for 52 weeks before having the opportunity to move into the open-label extension (OLE). Currently, omaveloxolone is commercialized under the brand name SKYCLARYS® in over 40 countries, including in the U.S. and the European Union, and is the only approved product for FA in adults and adolescents aged 16 years and older.