NDAINTRATHECALSOLUTIONPriority Review
Approved
Apr 2023
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
8
Data completeness
4/8 — Partial
Priority Review
Drug data last refreshed 1h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
QALSODY (tofersen) is an antisense oligonucleotide administered intrathecally for the treatment of SOD1-ALS (amyotrophic lateral sclerosis). It works by targeting and reducing SOD1 protein production in the central nervous system. This represents a precision therapy approach for a rare genetic form of ALS with limited treatment options.
$0.454MPart D
Peak8.8 years to LOEEarly-stage peak lifecycle with minimal claims volume (26 in 2023) indicates a niche orphan indication with limited addressable market and small commercial team footprint.
Mechanism of Action
Pharmacologic Class:
Antisense Oligonucleotide
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (8)
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 Jan 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
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.