What is Eteplirsen?
Eteplirsen helps the body produce a dystrophin protein. Dystrophin, a type of protein that helps stabilize muscle tissue structure and function properly, is essential for muscle growth and development. Muscle fibers become weaker and more easily damaged without dystrophin. Walking, for example, can be affected by this.A mutation in a gene is responsible for Duchenne muscle dystrophy. This would cause dystrophin to be produced by the body.Eteplirsen can be used to treat children who have Duchenne muscle dystrophy. Eteplirsen does not cure DMD, but it may reduce muscle weakness or muscle wasting.The FDA approved Eteplirsen on an "accelerated basis". Some children showed a positive response to the medicine in clinical trials. Further studies will be needed to see if eteplirsen can treat DMD in general.Eteplirsen can be used in other ways not mentioned in the medication guide.
Side effects of Eteplirsen
If your child shows any of the following symptoms of an allergy reaction, seek immediate medical attention: difficulty breathing or swelling in your lips, face, throat, or tongue.
Some of the most common side effects associated with eteplirsen include:
- Pain, swelling, bruising, or skin rash where the medication was injected.
- Vomiting;
- Joint Pain
- Problems with balance
- Symptoms of a cold, including a stuffy nose and a sore throat.
There may be other side effects. For medical advice on side effects, call your doctor.Report side effects by calling the FDA at 1-800-FDA-1088.
Warnings
Tell the doctor all of your child's allergies and medical conditions before your child is prescribed eteplirsen.
Before you take this drug
Inform your doctor of any severe reactions your child has experienced.Tell the doctor all of your child's allergies and medical conditions before your child is prescribed eteplirsen.It is unknown if eteplirsen can be harmful to a woman who is pregnant or nursing.
How to take Eteplirsen?
Eteplirsen can be infused into the vein. This injection will be given by a healthcare professional.A numbing agent can be used to make the IV injections more comfortable.Eteplirsen should be administered once a week. The infusion of this medicine can last up to an hour.When an eteplirsen injection is administered, tell your healthcare provider if you experience chest pain, tightness, and/or difficulty breathing.Inform your doctor of any weight changes. Eteplirsen dosages are determined by weight. Any changes in weight may change the dose.
Details on dosage
Adult dose for muscle dystrophy:
Infuse 30 mg/kg intravenously once per week over 35–60 minutes.
Comments:
Use a 0.2-micron in-line filter during infusion.
This drug has been approved for accelerated approval due to an increased dystrophin level in the skeletal muscles of some patients. A clinical benefit, however, has yet to be established.
The approval of a drug may depend on the clinical benefits of follow-up trials.
Use: To treat Duchenne muscular dystrophy in patients with a mutation that allows exon 51 to be skipped.
The usual pediatric dose for muscular dystrophy
Infuse 30 mg/kg intravenously once per week over a period of 35–60 minutes.
Comments:
Use a 0.2-micron in-line filter during infusion.
This drug has been approved for accelerated approval due to an increased dystrophin level in the skeletal muscles of some patients. A clinical benefit, however, has yet to be established.
The approval of a drug may depend on the clinical benefits of follow-up trials.
Use: To treat Duchenne muscular dystrophy in patients with a mutation that allows exon 51 to be skipped.
What happens if I miss the dose?
If you are unable to attend your child’s appointment, call your doctor.
What happens if I overdose?
Call 1-800-222-1222 for poison help or seek immediate medical attention.
What should be avoided?
If your doctor prescribes any restrictions on foods, drinks, or activities, follow their instructions.
Interaction with other drug
Other medications, such as prescription drugs, over-the-counter products, vitamins, and herbal remedies, may also affect eteplirsen. Inform your child's health care providers of all the medicines he or she is taking, as well as any new medications.
