NDAINTRATHECALSOLUTIONPriority Review
Approved
Apr 2023
Lifecycle
Growth
Competitive Pressure
0/100
Clinical Trials
8
Mechanism of Action
Pharmacologic Class:
Antisense Oligonucleotide
Clinical Trials (5)
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
Started Mar 2026
125 enrolled
Amyotrophic Lateral Sclerosis
Tofersen in Non-SOD1 ALS
Started Dec 2025
30 enrolled
ALS (Amyotrophic Lateral Sclerosis)
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
Started Dec 2025
12 enrolled
Amyotrophic Lateral Sclerosis
A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Started May 2021
158 enrolled
Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
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
Started Dec 2018
8 enrolled
Healthy Volunteers
Loss of Exclusivity
LOE Date
Apr 1, 2035
110 months away
Patent Expiry
Apr 1, 2035
Exclusivity Expiry
Apr 25, 2030