Vyondys 53

About Golodirsen 

• Golodirsen is an antisense oligonucleotide belonging to the family called Morpholino antisense Oligomers. 
• Golodirsen is used for the treatment of Duchenne Muscular Dystrophy (DMD) in patients with a verified mutation in the DMD gene that can be addressed through exon 53 skipping. 
• Exon 53 skipping represents a form of gene therapy employing antisense oligonucleotides to obstruct the generation of dystrophin protein that lacks exon 53, permitting the body to produce a truncated yet functional dystrophin protein. 
• The accelerated approval of this indication has been granted based on the observed increase in dystrophin production within skeletal muscle in patients treated with Golodirsen. 
• To maintain this approval, it may be necessary to confirm and elucidate the clinical benefits through a subsequent confirmatory trial.
• This treatment is typically administered under the supervision of a healthcare professional and may require regular injections or infusions. It’s important to consult with a healthcare provider for more specific information and guidance regarding the process. 

Strength: 

Injection: IV infusion of 100mg/2mL (50mg/mL) in a single-dose vial.

Recommended Dosage:

The drug is administered by a health care professional once a week directly into the bloodstream through intravenous (a vein) or IV infusion. It takes approximately an hour for Golodirsen to be infused into the body. Before beginning the treatment, it is advisable to assess the levels of serum cystatin C, urine protein-to-creatinine ratio, and urine dipstick. 

• 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 Golodirsen for your individual needs should be discussed with a healthcare professional.

Important:

Discontinuing the treatment should only occur under the guidance of your physician. Importantly, this medication should not be considered a replacement for standard care. Individuals using Golodirsen 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 Golodirsen, it’s crucial to provide a comprehensive medical history, which includes any allergies, infections, or existing medical conditions. Make sure to disclose all medications you are currently taking, including over-the-counter drugs and herbal supplements. 

Warnings & Precautions

• Golodirsen can trigger a severe allergic reaction known as hypersensitivity reactions. Patients must undergo close monitoring for anaphylactic signs and symptoms during and after each infusion and discontinuation is needed if such reactions occur. 
• The hypersensitivity reaction includes, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in patients who were treated with Golodirsen.
• Golodirsen has been associated with kidney toxicity in animal studies, and although clinical studies didn’t show kidney toxicity. 
• Limited clinical experience exists, and kidney issues have been observed with some antisense oligonucleotides. Therefore, kidney function should be monitored in patients using Golodirsen, especially in patients with pre-existing kidney disease.
• Golodirsen is not recommended for pregnant and breastfeeding women unless it is advised by a doctor. Because, the safety and efficacy is unknown in such population. 
• Golodirsen has not been specifically studied in patients with liver impairment. However, patients with hepatic impairment may be at increased risk for developing hepatotoxicity.
• Golodirsen should be used with caution in patients taking other medications that may interact with the current medicine.

Common Golodirsen  Side Effects:

• Headache
• Pyrexia 
• Abdominal pain
• Nasopharyngitis
• Cough
• Vomiting
• Rash
• Nausea
• Pain at the injection site

Use in Specific Population

• Golodirsen 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.
• Golodirsen 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 Golodirsen 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 Golodirsen 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.