Drug profile · INN
eteplirsen
EXONDYS
eteplirsen (EXONDYS) is a dystrophin pre-mrna positive modulator. Associated with Sarepta Therapeutics. Route of administration: intravenous. Current US regulatory status: approved.
Drug details — eteplirsen
- US status
- approved
- Class
- Musculo-skeletal system (M09)
- Route
- INTRAVENOUS
- Patents
- 20
- Programs
- 8
- Data quality
- 0.89
Quick answer
eteplirsen (EXONDYS) is a dystrophin pre-mrna positive modulator. Associated with Sarepta Therapeutics. Route of administration: intravenous. Current US regulatory status: approved.
Key facts
Mechanism of action
Dystrophin pre-mRNA positive modulator
Primary target: Dystrophin pre-mRNA
Approval history
| Authority | Status | Date |
|---|---|---|
| FDA | approved | — |
| EMA | refused | — |
Pipeline programs
Related & competitor drugs
Related patents
-
US 12239715
Peptide oligonucleotide conjugates
granted -
US 11732259
Peptide oligonucleotide conjugates
granted -
US 11674139
Oligonucleotides for treating expanded repeat diseases
granted -
US 11672871
Peptide oligonucleotide conjugates
granted -
US 11225662
Peptide oligonucleotide conjugates
granted -
US 11097011
Peptide oligonucleotide conjugates
granted -
US 11071749
Oligonucleotide analogues targeting human LMNA
granted -
US 10675356
Peptide oligonucleotide conjugates
granted -
US 10577605
Induced exon inclusion in spinal muscle atrophy
granted -
US 10487326
Peptide oligonucleotide conjugates
granted -
US 10364431
Compositions for treating muscular dystrophy
granted -
US 10337003
Compositions for treating muscular dystrophy
granted -
US 10076536
Oligonucleotide analogues targeting human LMNA
granted -
US 10066228
Oligonucleotides for treating expanded repeat diseases
granted -
US 9944926
Induced exon inclusion in spinal muscle atrophy
granted -
US 9920085
Boronic acid conjugates of oligonucleotide analogues
granted -
US 9862946
Peptide oligonucleotide conjugates
granted -
US 9682097
Oligonucleotide analogues targeting human LMNA
granted -
US 9506058
Compositions for treating muscular dystrophy
granted -
US 9453225
Multiple exon skipping compositions for DMD
granted
Frequently asked questions
What is eteplirsen?
eteplirsen (EXONDYS) is a dystrophin pre-mrna positive modulator. Associated with Sarepta Therapeutics. Route of administration: intravenous. Current US regulatory status: approved.
What is the brand name for eteplirsen?
EXONDYS is a marketed brand name for eteplirsen. Check FDA Drugs@FDA for the current US label holder and prescribing information.
What is the US approval status of eteplirsen?
eteplirsen has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.