NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Aug 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
3
Data completeness
3/8 — Basic
Priority Review
Drug data last refreshed 10h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VILTEPSO (viltolarsen) is an intravenous antisense oligonucleotide approved for Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon-skipping therapy. It works by modulating dystrophin protein expression through antisense mechanism. The drug targets a rare, severe genetic muscle-wasting disease with high unmet medical need.
Peak7.8 years to LOE
As a peak-stage rare disease IV biologic with no direct competition, the brand team is focused on market penetration and patient identification in a small, specialized neurology segment.
Mechanism of Action
Mechanism of action data is being enriched from DailyMed and FDA sources. Check back soon for updated drug intelligence.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (3)
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
Started Jul 2021
20 enrolled
Duchenne Muscular Dystrophy
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
Started Apr 2021
74 enrolled
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
Started Apr 2020
77 enrolled
Duchenne Muscular Dystrophy
Career Relevance
Working on VILTEPSO offers exposure to orphan drug commercialization, rare disease market dynamics, and highly specialized neurology networks with deep patient relationships. The niche market and peak lifecycle stage create stable but limited growth opportunities, ideal for professionals seeking focused expertise in ultra-rare disease rather than mass-market scale.
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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.