1 INDICATIONS AND USAGE
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
6 ADVERSE REACTIONS
6.1 Clinical Trials Experience
All patients were male and had genetically confirmed Duchenne muscular dystrophy.
8 USE IN SPECIFIC POPULATIONS
8.4 Pediatric Use
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, including pediatric patients.
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3.
14 CLINICAL STUDIES
EXONDYS 51 was evaluated in three clinical studies in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.