NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Feb 2021
Lifecycle
Peak
Competitive Pressure
8/100
Clinical Trials
4
Data completeness
5/8 — Partial
Priority Review
AMONDYS 45 (casimersen) by Sarepta Therapeutics is 45 of dystrophin pre-mrna resulting in exclusion of this exon during mrna processing in patients with genetic mutations that are amenable to exon 45 skipping. First approved in 2021.
Drug data last refreshed 12h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
AMONDYS 45 (casimersen) is an antisense oligonucleotide that induces skipping of exon 45 in dystrophin pre-mRNA, allowing production of an internally truncated but functional dystrophin protein. It is administered intravenously and is indicated for patients with Duchenne muscular dystrophy (DMD) who have genetic mutations amenable to exon 45 skipping. This represents a precision genetic therapy approach to a severe, progressive neuromuscular disorder.
Peak4.0 years to LOE
Product is at peak commercial maturity with stable positioning in a niche ultra-rare disease market; teams are likely focused on maximizing penetration within the small eligible patient population.
Mechanism of Action
45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that…
Pharmacologic Class:
Antisense Oligonucleotide
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (4)
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
Started Feb 2020
3 enrolled
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Started Jan 2019
300 enrolled
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Started Aug 2018
171 enrolled
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Started Sep 2016
228 enrolled
Duchenne Muscular Dystrophy
Worked on AMONDYS 45 at Sarepta Therapeutics? Share your interview experience or compensation data (+7 days Pro)
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.