EXONDYS 51 -500mg (Eteplirsen Vials) 500mg/10ml

EXONDYS 51 is specifically indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This condition is a progressive muscle-wasting disorder.

• Duchenne muscular dystrophy (DMD): EXONDYS 51 is designed to promote the production of a truncated but functional dystrophin protein, which can help mitigate the effects of the genetic mutation in DMD patients.

• Eteplirsen works by binding to exon 51 of the dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing. This exon skipping allows for the production of an internally truncated dystrophin protein, which is intended to be functional and provide clinical benefit in patients with DMD who have mutations amenable to exon 51 skipping.

EXONDYS 51, like all medications, can cause side effects. Common Side Effects:

• Application site reactions: These may include pain, swelling, or redness at the infusion site.
• Headache: Some patients may experience headaches during or after the infusion.
• Vomiting: Nausea and vomiting can occur as a side effect.
• Cough: Some patients may experience coughing during the infusion.
• Fever: An increase in body temperature may be observed.
• Fatigue: Patients may feel unusually tired or weak.

Serious or Rare Side Effects

• Hypersensitivity reactions: These can include symptoms such as rash, itching, or difficulty breathing. Immediate medical attention is required if these symptoms occur.
• Bronchospasm: Some patients may experience difficulty breathing or wheezing during the infusion.
• Cyanosis: A bluish discoloration of the skin due to lack of oxygen may occur in rare cases.

EXONDYS 51 should be used with caution and under the supervision of a healthcare professional. It is important to follow the prescribed dosage and administration instructions carefully. Precautions include:

• Hypersensitivity: Patients with a known hypersensitivity to eteplirsen or any of its components should not use this medication.
• Infusion reactions: Monitor patients closely during and after infusion for signs of hypersensitivity reactions or infusion-related reactions.
• Renal impairment: The safety and efficacy of EXONDYS 51 in patients with renal impairment have not been established.
• Pregnancy: The safety of EXONDYS 51 during pregnancy has not been established. Consult a healthcare provider before use.
• Breastfeeding: It is not known whether eteplirsen is excreted in human milk. Consult a healthcare provider before use.
• Store at 2-8°C: Keep refrigerated, protect from light, do not freeze or shake.

EXONDYS 51 may interact with other medications. It is important to inform your healthcare provider about all medications you are taking. Major Interactions (Avoid):

• None identified.

Moderate Interactions (Monitor Closely)

• Other medications that may cause infusion-related reactions: Concurrent use of other medications that can cause infusion-related reactions should be monitored closely.
• Medications that affect renal function: Monitor renal function closely when using EXONDYS 51 with other medications that affect renal function.