Golodirsen
Indications
Golodirsen is used for:
Duchenne Muscular Dystrophy
Adult Dose
Duchenne Muscular Dystrophy
Indicated for Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping
30 mg/kg IV qWeek
Child Dose
Renal Dose
Renal impairment
Renal clearance is reduced in non-DMD adults with renal impairment, based on estimated glomerular filtration rate (eGFR) calculated using the Modification of Diet and Renal Disease (MDRD) equation
No specific dosage adjustment can be recommended for patients with DMD who have renal impairment based on eGFR
Closely monitor patients with known renal function impairment during treatment
Administration
Contra Indications
Precautions
Hypersensitivity
Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation, reported, some requiring treatment
If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting therapy
Renal toxicity
Renal toxicity was observed in animals
Although renal toxicity was not observed in the clinical studies, renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides
Monitor renal function during therapy
Pregnancy-Lactation
Pregnancy
No human or animal data are available to assess use during pregnancy
Lactation
No human or animal data are available to assess effects on milk production, presence in milk, or effects on the breastfed infant
Interactions
Adverse Effects
Side effects of Golodirsen :
>10%
Headache (41%)
Pyrexia (41%)
Fall (29%)
Abdominal pain (27%)
Nasopharyngitis (27%)
Cough (27%)
Vomiting (27%)
Nausea (20%)
1-10%
>5%
Administration site pain
Back pain
Pain
Diarrhea
Dizziness
Ligament sprain
Contusion
Influenza
Oropharyngeal pain
Rhinitis
Skin abrasion
Ear infection
Seasonal allergy
Tachycardia
Catheter site-related reaction
Constipation
Fracture
Frequency Not Defined
Hypersensitivity
Mechanism of Action
Binds to exon 53 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping
Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping