Tofersen
- Generic Name
- Tofersen
- Brand Names
- Qalsody
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 2088232-70-4
- Unique Ingredient Identifier
- 2NU6F9601K
- Background
Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented. Tofersen was granted accelerated approval from the FDA on April 25, 2023,...
- Indication
Tofersen is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on the reduction in plasma neurofilament light chain (NfL) observed in patients treated with tofersen. Continued approval for this indication may b...
- Associated Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Associated Therapies
- -
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
- First Posted Date
- 2021-07-22
- Last Posted Date
- 2024-06-10
- Lead Sponsor
- Biogen
- Registration Number
- NCT04972487
- Locations
- 🇺🇸
Research Site, Austin, Texas, United States
A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
- Conditions
- Interventions
- First Posted Date
- 2021-04-23
- Last Posted Date
- 2024-06-11
- Lead Sponsor
- Biogen
- Target Recruit Count
- 150
- Registration Number
- NCT04856982
- Locations
- 🇺🇸
Emory University, Atlanta, Georgia, United States
🇺🇸Northwestern University, Chicago, Illinois, United States
🇺🇸Johns Hopkins Hospital, Baltimore, Maryland, United States
A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults
- First Posted Date
- 2018-12-05
- Last Posted Date
- 2023-06-22
- Lead Sponsor
- Biogen
- Target Recruit Count
- 8
- Registration Number
- NCT03764488
- Locations
- 🇺🇸
Research Site, New York, New York, United States
Long-Term Evaluation of BIIB067 (Tofersen)
- Conditions
- Interventions
- First Posted Date
- 2017-03-03
- Last Posted Date
- 2024-08-19
- Lead Sponsor
- Biogen
- Target Recruit Count
- 139
- Registration Number
- NCT03070119
- Locations
- 🇬🇧
Research Site, Sheffield, South Yorkshire, United Kingdom
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
- First Posted Date
- 2015-12-08
- Last Posted Date
- 2023-07-28
- Lead Sponsor
- Biogen
- Target Recruit Count
- 176
- Registration Number
- NCT02623699
- Locations
- 🇺🇸
Emory University Hospital, Atlanta, Georgia, United States
🇨🇦Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
Drug Development Updates
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