APPROVED_FOR_MARKETINGNot Applicable

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

United States

Plain-language summary

The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.

Who can participate

Age range18 Years

SexALL

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Key Inclusion Criteria: * Medically able to undergo the program procedures, as determined by the treating healthcare professional (HCP). * Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS). Key Exclusion Criteria: * Previous or current participation in a clinical trial of tofersen. * Use of an investigational medicinal product (IMP) for amyotrophic lateral sclerosis (ALS) within 5 half-lives of the IMP before the first dose of tofersen. * Participant's primary place of residence is outside of the country of treatment. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Trial details

NCT IDNCT04972487

SponsorBiogen

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

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