Trial Profile
A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation
Status:
Completed
Phase of Trial:
Phase III
Latest Information Update: 09 Mar 2025
Price :
$35
*
At a glance
- Drugs Tofersen (Primary)
- Indications Amyotrophic lateral sclerosis
- Focus Adverse reactions; Pharmacokinetics; Proof of concept; Registrational; Therapeutic Use
- Acronyms VALOR
- Sponsors Biogen
- 04 Mar 2025 According to a Biogen media release, the authorization is conditional, pending the results of trials to verify its clinical benefit. The final OLE clinical report, summary of integrated analyses with VALOR trial, and results of the ongoing Phase 3 ATLAS study of tofersen in clinically presymptomatic SOD1-ALS patients will serve as the confirmatory trials.
- 04 Mar 2025 According to a Biogen media release, based on acceptable safety profile, high quality and promising efficacy of QALSODY from the VALOR trial, Health Canada has issued marketing authorization with conditions (Notice of Compliance with Conditions (NOC/c)) for QALSODY (tofersen injection) for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase (SOD1) gene.
- 30 May 2024 According to a Biogen media release, the European Commission (EC) has granted marketing authorization under exceptional circumstances and maintained orphan designation for QALSODY(tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS), based on results form this trial.