Eteplirsen, or Exondys 51, is the first FDA-approved treatment for Duchenne patients with exon 51 removal.
To use Exondys 51, a person must have a mutation in the DMD gene that can be treated by “jumping” from Exon 51. Exons are part of a gene. Exon 51 refers to a specific exon in the DMD gene.
Exondys 51 is an intravenous injection that is given over a period of time. Exondys 51 is available in one dose: 50 mg / ml (mg / ml). The active drug in Exondys 51 is eteplirsen. Eteplirsen is a class of drugs known as antisense oligonucleotides.
Exons are part of a gene. The missing exon causes a mistake in the DMD gene instructions for making dystrophin, and this leads to a lack of sufficient dystrophin in the body.
Exondys 51 has a unique mechanism of action. It causes you to skip reading Exon 51 while reading the genetic instructions for making dystrophin. This causes the body to produce a shorter form of dystrophin that is still effective.
Exondys 51 reaches its highest level in the body immediately after receiving it. But because of how the medicine works, you probably do not feel that it works. But it does not start to increase dystrophin levels in the body so soon. It usually takes 48 weeks for the drug to work properly.