The objective of this early access program (EAP) is to provide access to tofersen toeligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutationin the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism inorder to address a high unmet medical need in this population.
Treating healthcare professionals (HCPs) of participants who meet the inclusion/exclusion
criteria may request access by emailing medicineaccess@clinigengroup.com. The current EAP
structure does not limit participants to receive access at specific sites. Treating HCPs
can submit requests on behalf of participants by emailing Clinigen at the email address
provided below. Information on the EAP eligibility criteria, including participants and
center criteria, will be provided by Clinigen as part of the enrollment request process.
Drug: Tofersen
Administered as intrathecal bolus injection
Other Name: BIIB067,QALSODY
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
Research Site
Anchorage, Alaska, United States
Research Site
Los Angeles, California, United States
Research Site
Orange, California, United States
Research Site
San Diego, California, United States
Research Site
San Francisco, California, United States
Research Site
Chicago, Illinois, United States
Research Site
Kansas City, Kansas, United States
Research Site
Boston, Massachusetts, United States
Research Site
Worcester, Massachusetts, United States
Research Site
Ann Arbor, Michigan, United States
Research Site
Detroit, Michigan, United States
Research Site
Rochester, Minnesota, United States
Research Site
Saint Louis, Missouri, United States
Research Site
Lebanon, New Hampshire, United States
Research Site
Amherst, New York, United States
Research Site
New York, New York, United States
Research Site
Syracuse, New York, United States
Research Site
Philadelphia, Pennsylvania, United States
Research Site
Austin, Texas, United States
Medical Director, Study Director
Biogen