What is Eteplirsen?
Eteplirsen is used to treat a certain inherited muscle disorder (Duchenne muscular dystrophy-DMD). This disorder is caused by a lack of a certain muscle protein (dystrophin). This medication can increase the amount of dystrophin in your body.
Is eteplirsen FDA approved?
On September 19, 2016, FDA finally approved eteplirsen for Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular degenerative disorder that causes severe muscle loss and premature death.
What type of drug is Eteplirsen?
Eteplirsen is an antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer.
Is eteplirsen a cure?
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 only targets specific mutations and can be used to treat about 14% of DMD cases.
What are the two side effects of eteplirsen?
The recommended regimen is 30 mg per kg body weight once weekly by intravenous infusion. Side effects of eteplirsen include headache, fever, falls, abdominal pain, cough and nausea. Injection site reactions and hypersensitivity reactions including rash, pruritus, urticaria and skin exfoliation have occurred.
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.
How does Exondys 51 work?
A lack of dystrophin causes muscle cells to become damaged and weakened over time. EXONDYS 51 uses exon-skipping technology to allow the body to make a shorter form of the dystrophin protein in some patients by skipping over a specific exon on the dystrophin gene.
What are two side effects of eteplirsen?
Is eteplirsen approved in Europe?
A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe.
How much is Eteplirsen?
Exondys 51 (eteplirsen) – between $750,000 and $1.5m a year DMD is a rare progressive disease caused due to one of more than 2,000 mutations of the dystrophin gene.
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.
What are the 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.