Golodirsen

Golodirsen Brand Name– Vyondys

What is Golodirsen

Golodirsen is an injectable antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

Approximately 8% of the DMD population has this mutation.

DMD is a rare, X-chromosome-linked disease characterized by progressive muscle deterioration and weakness; it affects approximately 1 in 3,500 to 5,000 male births.

Affected children typically develop symptoms in early childhood that quickly progress to a loss of ambulation in adolescence and ultimately, life-threatening heart and respiratory conditions that limit life expectancy to early adulthood.

FDA-approval was based on the surrogate endpoint of increased dystrophin production in skeletal muscle, which the FDA has stated is reasonably likely to predict clinical benefit (e.g., improved motor function).

During a small 2-part clinical study (n = 25), mean dystrophin concentrations increased from 0.1% of normal at baseline to 1.02% of normal after 48 weeks of golodirsen treatment.

The most common adverse reactions reported include headache, fever, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. Measurement of glomerular filtration rate prior to initiation and monitoring for renal toxicity during treatment is recommended.

Although not observed in clinical trials, renal toxicity has been observed in golodirsen-treated animals and in humans after administration of some antisense oligonucleotides

Indications

  • Duchenne muscular dystrophy

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

NOTE: Golodirsen is designated an orphan drug by the FDA for treatment of Duchenne muscular dystrophy.

Side Effects

  1. abdominal pain
  2. back pain
  3. constipation
  4. cough
  5. diarrhea
  6. dizziness
  7. exfoliative dermatitis
  8. fever
  9. headache
  10. influenza
  11. injection site reaction
  12. nausea
  13. pharyngitis
  14. pruritus
  15. rash
  16. rhinitis
  17. sinus tachycardia
  18. urticaria
  19. vomiting

Monitoring Parameters

  • 24-hour urinary protein
  • glomerular filtration rate
  • serum cystatin C
  • urinalysis

Contraindications

  • breast-feeding
  • geriatric
  • pregnancy
  • renal disease
  • renal failure
  • renal impairment

Interactions

There are no drug interactions associated with Golodirsen products.

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