What is Vyondys 53 (golodirsen) for?

Vyondys 53 (golodirsen) is an antisense nucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.^[1]

It is available in vial form containing 100 mg/2mL golodirsen.^[1]

How does Vyondys 53 (golodirsen) work?

Duchenne muscular dystrophy (DMD) is one of the most common lethal progressive muscle-wasting diseases that is occurring primarily in young boys. In patients with DMD, the protein dystrophin is not functioning properly. Dystrophin is crucial for muscle health, and dysfunctionality of it leads to muscle weakness and loss of muscles.^[2]

This lack of functional dystrophin is caused by a mutation in the dystrophin gene. Most genes are composed of alternating ‘exons’, which contain a genetic code for the production of proteins, and ‘introns’, which do not contain a code. To produce a protein out of this genetic code, the cells make a temporary copy of the gene, called messenger RNA (mRNA). Subsequently, this mRNA will be ‘spliced’, in which the intron regions will be cut out of the mRNA and exons will be fused together, finalizing the complete code for the protein.^[3]

In some cases of DMD, certain parts of the DMD gene are not present, which causes the exons to not fit together properly. This disrupts the code, and leads to the production of a non-functional dystrophin protein.^[3]

Vyondys 53 (golodirsen) is an antisense oligonucleotide. It contains a piece of artifcial mRNA that restores this disruption by fitting the exons together again. Golodirsen is designed specifically for exon 53 of the DMD gene, so it is only effective in patients with a mutation that is amenable to exon 53 skipping.^[1]

Where has Vyondys 53 (golodirsen) been approved?

Vyondys 53 (golodirsen) was approved for the treatment of DMD patients with a confirdmed mutation of the DMD gene that is amenable to exon 53 skipping by:

• The Food and Drug Administration (FDA), USA, on December 12, 2019.^[4]

The FDA approved Vyondys 53 (golodirsen) under accelerated approval.^[4]

Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.

How is Vyondys 53 (golodirsen) taken?

The standard dosage is:^[1]

• 30 mg/kg administered as an intravenous infusion over 35-60 minutes once weekly.

Complete information about Vyondys 53 (golodirsen) dosage and administration can be found in the official prescribing information listed in our references section.^[1]

Note: Please consult with your treating doctor for personalised dosing.

Are there any known adverse reactions or side effects of Vyondys 53?

Common adverse reactions

The most common adverse reactions (≥20% of patients) listed in the prescribing information include:^[1]

• Headache
• Loss of appetite (pyrexia)
• Fall
• Abdominal pain
• Nasopharyngitis
• Cough
• Vomiting
• Nausea

Serious adverse reactions

The serious adverse reactions listed in the prescribing information include:^[1]

• Renal toxicity, including potentially fatal glomerulonephritis (kidney inflammation)

Use in a specific population

It is not yet known if Vyondys 53 (golodirsen) affects an unborn baby.^[1]

For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information.