What type of drug is Eteplirsen?
Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation….Eteplirsen.
| Clinical data | |
|---|---|
| Drug class | Antisense oligonucleotide |
| ATC code | M09AX06 (WHO) |
| Legal status | |
| Legal status | US : ℞-only |
What is Eteplirsen?
What is eteplirsen? Eteplirsen works by helping the body produce a protein called dystrophin. Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged.
Is eteplirsen a gene therapy?
Eteplirsen is an exon-skipping therapeutic. Notes: Eteplirsen (green bar) specifically recognizes exon 51 of the DMD gene. Upon binding, it influences the splicing machinery to skip exon 51 from the mature mRNA transcript.
Is Exondys 51 gene therapy?
Exondys 51 is approved by the Food and Drug Administration (FDA) to treat DMD in people who have a certain gene mutation. “Gene mutation” means that a specific gene isn’t working the way it should. To use Exondys 51, you must have a mutation in the DMD gene that can be treated by “skipping” exon 51.
What is Eteplirsen used for?
Eteplirsen (e TEP ler sun) is a drug which lets dystrophin, the muscle protein missing in Duchenne muscular dystrophy, partially work. This medicine is used for the treatment of Duchenne muscular dystrophy in patients who have a specific gene mutation.
When was eteplirsen approved?
Development Timeline for Exondys 51
| Date | Article |
|---|---|
| Sep 19, 2016 | Approval FDA Approves Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy |
| Jun 6, 2016 | Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA |
What are the two side effects of eteplirsen?
Common side effects may include:
- pain, bruising, itching, skin rash, or other irritation where the medicine was injected;
- vomiting;
- joint pain;
- problems with balance; or.
- cold symptoms such as stuffy nose, sneezing, sore throat.
Is eteplirsen FDA approved?
FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD).
Can a geneticist tell which exon is missing?
Your child’s doctor or genetic counselor will determine amenability to exon 51 skipping based on which exons are missing. If the genetic test report mentions deletions, it will also identify the “genomic region” or simply add a range of missing exons (e.g., 48-50).
What are two side effects of eteplirsen?
Common side effects of Exondys 51 include:
- balance disorder,
- vomiting,
- skin rash,
- bruising,
- joint pain,
- catheter site pain, and.
- upper respiratory tract infection.
How much does Exondys 51 cost?
Developed by Sarepta Therapeutics, EXONDYS 51® is estimated to cost between $750,000 and $1.5 million a year.
How much does eteplirsen cost?
Exondys 51 (eteplirsen) – between $750,000 and $1.5m a year EXONDYS 51® is approved for the treatment of Duchenne muscular dystrophy.