Exondys 51

About Exondys 51

• Exondys 51 is an antisense oligonucleotide used to treat Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 53 skipping. 
• Exon 53 skipping is a gene therapy approach using antisense oligonucleotides to prevent the production of dystrophin protein lacking exon 53, allowing the body to produce a shorter but functional dystrophin protein. 
• Accelerated approval was granted based on increased dystrophin production in skeletal muscle of treated patients. Continued approval may hinge on confirming clinical benefits through subsequent trials. 
• Treatment involves healthcare professional supervision, potentially requiring regular injections or infusions. Consult a healthcare provider for specific guidance.

Strength: 

Injection: 100mg/2mL (50mg/mL); 500mg/10mL (50mg/mL) in single-dose vial.

Recommended Dosage:

The drug is administered by a health care professional once a week directly into the bloodstream through intravenous or IV infusion. It takes approximately an hour for Exondys 51 to be infused into the body. Contact your doctor or seek emergency medical attention at the nearest hospital.

• A weekly dose of  30mg/kg of body weight is recommended for. As an intravenous infusion, it takes 35 to 60 minutes and is injected through an inline 0.2 micron filter. 
• Patients may need to adjust the dosage of medication according to their response and side effects.
• The recommended dosage of Exondys 51 for your individual needs should be discussed with a healthcare professional. 

Important:

Discontinuing Exondys 51 treatment should be done only under the guidance of your physician. It’s essential to understand that this medication is not a replacement for standard care. Individuals using Exondys 51 should continue to receive all recommended treatments, such as physical therapy and occupational therapy. If you have any questions or concerns, please don’t hesitate to discuss them with your healthcare provider.

To ensure your safe and effective use of Exondys 51, it’s crucial to provide a comprehensive medical history, including any allergies, infections, or existing medical conditions. Additionally, disclose all medications you are currently taking, including over-the-counter drugs and herbal supplements. If you have questions or concerns about this medication, discuss them with your doctor. Following these instructions diligently is essential to maximize the medicine’s benefits and minimize the risk of side effects.

Warnings & Precautions

• Exondys 51 may trigger severe allergic reactions, known as hypersensitivity reactions, which can include symptoms such as rash, pyrexia (fever), pruritus (itching), urticaria (hives), dermatitis, and skin exfoliation. Close monitoring for signs of anaphylaxis during and after each infusion is essential, and discontinuation of Exondys 51 may be necessary if such reactions occur.
• Although clinical studies did not show kidney toxicity, Exondys 51 has been associated with kidney toxicity in animal studies. Limited clinical experience exists, so kidney function should be monitored, especially in patients with pre-existing kidney disease.
• Exondys 51 is not recommended for pregnant and breastfeeding women unless advised by a doctor due to unknown safety and efficacy in these populations.
• Exondys 51 has not been specifically studied in patients with liver impairment. Patients with hepatic impairment should use Exondys 51 with caution as they may be at increased risk for developing hepatotoxicity.
• Exondys 51 should be used cautiously in patients taking other medications that may interact with it. Please consult your healthcare provider for guidance on potential interactions and medication management.

Common Exondys 51 Side Effects:

• Headache
• Pyrexia 
• Abdominal pain
• Nasopharyngitis
• Cough
• Vomiting
• Rash
• Nausea
• Pain at the injection site
• Dyspnea
• Bronchospasm
• Urticaria
• Hypotension

Use in Specific Population

• Exondys 51 has not been studied in pregnant or lactating women, so there is no way to know if it is safe or effective for these populations. If you are pregnant or breastfeeding, talk to your doctor about the risks and benefits of the medicine.
• Exondys 51 can be given to children as young as 6 months old, but it hasn’t been studied in older adults. However, older people might be more prone to certain side effects, like reactions during the infusion and potential liver issues.

Storage and Handling

• Store Exondys 51 at temperatures between 2°C to 8°C (36°F to 46°F) and ensure it does not freeze.
• Avoid shaking the vial and protect it from exposure to light. 
• Remember that Exondys 51 is a sterile product, so do not use it if the vial is damaged or the seal is broken. 
• Always employ aseptic techniques and discard any unused portions. Never reuse the vial or infusion set to maintain safety and effectiveness.