QALSODY (Biogen Australia Pty Ltd)
Product name
QALSODY
Sponsor
Date registered
Evaluation commenced
Decision date
Approval time
239 (255 working days)
Active ingredients
tofersen
Registration type
NCE/NBE
Indication
QALSODY has provisional approval in Australia for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. The decision to approve this indication has been made on the totality of evidence including impacts on clinical function, respiratory function, muscle strength, survival, target engagement and reductions in NfL (a marker of axonal injury and neurodegeneration) in SOD1-ALS patients treated with QALSODY. Continued approval of this indication depends on additional data.
Registration process
Orphan drug
Sponsors receive a fee waiver to help bring medicines for a small population to market.