Eteplirsen (Exondys 51) is the first treatment that has been approved for the treatment of specific types of Duchenne muscular dystrophy. It is an antisense oligonucleotide that targets exon 51 of mRNA resulting in its exclusion during further processing. It is therefore only indicated in patients with a confirmed mutation of the DMD gene.

Eteplirsen (Exondys 51)Uses:

• Duchenne muscular dystrophy:

  • It is indicated for the treatment of patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

Eteplirsen (Exondys 51) Dose in Adults

Eteplirsen (Exondys 51) Dose in the treatment of Duchenne muscular dystrophy:

• 30 mg/kg intravenously once a week.

Eteplirsen (Exondys 51) Dose in Childrens

Eteplirsen (Exondys 51) Dose in the treatment of Duchenne muscular dystrophy (DMD):

• Male children older than 7 years of age:

  • 30 mg/kg/dose intravenously once a week.
  • In clinical trials, most of the candidates aged between 7 and 13 years.

Pregnancy Risk Category: N

• It has not yet been tested in pregnant women or in animal reproduction studies.

Eteplirsen use during breastfeeding:

• It is unknown if the drug will be excreted into breastmilk.
• Manufacturers recommend that you weigh the risks and benefits for the mother and infant.
• It has not yet been tested in breastfeeding females.

Dose in Kidney Disease:

• It has not been studied in patients with kidney disease.
• The manufacturer has not recommended any adjustment in the dose in patients with kidney disease.

Dose in Liver disease:

It has not been studied in patients with liver disease. The manufacturer has not recommended any adjustment in the dose in patients with liver disease.

Side Effects of Eteplirsen (Exondys 51):

• Central nervous system:

  • Equilibrium disturbance

• Dermatologic:

  • Contact dermatitis

• Gastrointestinal:

  • Vomiting

Contraindications to Eteplirsen (Exondys 51):

There are no contraindications for its use, according to the manufacturer.

Warnings and precautions

• Hypersensitivity

  • During or after the infusion, serious allergic reactions can occur.
  • Itching, rash and shortness of breath can all be symptoms of allergic reactions.
  • If there is a reaction, the infusion rate may be slowed down or stopped.

Monitoring Parameters:

Monitor for hypersensitivity reactions.

How to administer Eteplirsen (Exondys 51)?

• It is indicated for intravenous use only.
• The drug is administered by an intravenous infusion over 35 - 60 minutes.
• The infusion must be completed within four hours of the dilution.
• If the patient develops a hypersensitivity reaction, the rate of the infusion must be reduced.
• The IV access must be flushed with NS before and after the infusion.
• The infusion must not be infused with other medications and it should not be mixed with any other intravenous medicines.
• In case a dose is missed, it should be administered as soon as possible.

Mechanism of action of Eteplirsen (Exondys 51):

• It is an antisense nucleotide which binds to exon 51 of dystrophin pre–messenger RNA (mRNA).
• This results in it being exempt from further processing. 
• This allows the production of a dystrophin protein that is internally truncated.

Protein binding:

• 6% to 17%

Half-life elimination:

• 3 to 4 hours

Time to peak:

• 1.1 to 1.2 hours

Excretion:

• Renal

International Brand Names of Eteplirsen:

• Exondys 51

Eteplirsen Brand Names in Pakistan:

No Brands Available in Pakistan.