The FDA recently approved the use of Vamorolone, a novel corticosteroid, for the treatment of Duchenne muscular dystrophy in patients aged 2 years and above.
The drug enhances steroidal anti-inflammatory action and is a powerful antagonist against mineralocorticoid receptors. It is a safer and more effective alternative to glucocorticoids as it exhibits fewer side effects, such as weight gain, cushingoid appearance, growth retardation, and bone morbidities.
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Mechanism of action:
Glucocorticoids have numerous mechanisms of action that are responsible for both positive and negative effects.
Vamorolone is a dissociative steroid that only retains pathways that promote efficacy and does not activate pathways responsible for detrimental side effects.
Dosage and administration:
Dosage: 6 mg/kg once daily, patients weighing more than 50 kg may receive up to 300 mg daily.
Route of administration: directly dispensed into the mouth via a syringe.
Precautions for long-term use:
- Alterations in endocrine function
- Immunosuppression and increased risk of infection
- Alterations in cardiovascular/renal function
- Behavioral and mood disturbances.
References:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215239s000lbl.pdf
https://pubmed.ncbi.nlm.nih.gov136816559/
https://journals.plos.org/plosmedicine/article?id=10.1371/journal.pmed.1003222
