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Brand Name :
Vyondys 53
Synonyms :
golodirsen
Class :
Morpholino antisense oligomers, nucleic acids
Brand Name :
Vyondys 53
Synonyms :
golodirsen
Class :
Morpholino antisense oligomers, nucleic acids
Dosage Forms & Strengths
Concentrated solution, injection
100mg/2ml single vial dose
Dosage modification
Renal impairment
It is eliminated primarily by the kidneys, so individuals with renal impairment may not be able to clear the drug as effectively. In non-DMD adults with renal impairment, the dosage of golodirsen may need to be modified based on the individual's estimated glomerular filtration rate (eGFR) calculated using the Modification of Diet and Renal Disease (MDRD) equation. However, there is current:
30mg/kg intravenous every week
Dosage Forms & Strengths
Concentrated solution, injection
100mg/2ml single vial dose
Dosage modification
Renal impairment
It is eliminated primarily by the kidneys, so individuals with renal impairment may not be able to clear the drug as effectively. In non-DMD adults with renal impairment, the dosage of golodirsen may need to be modified based on the individual's estimated glomerular filtration rate (eGFR) calculated using the Modification of Diet and Renal Disease (MDRD) equation. However, there is current:
30mg/kg intravenous every week
Refer adult dosing
Actions and Spectrum
golodirsen targets and avoids exon 53 of the dystrophin gene, promoting RNA splicing and produces a functional protein called dystrophin. This helps to improve muscle action in DMD which is caused by exon 53 mutation.
Spectrum
golodirsen targets the with exon 53 mutations in DMD patients, but not other mutations of Duchenne Muscular Dystrophy.
Frequency defined:
>10%
Pyrexia
Abdominal pain
Cough
Nausea
Headache
Fall
Nasopharyngitis
Vomiting
1-10%
Back pain
Diarrhea
Ligament sprain
Influenza
Rhinitis
Ear infection
Tachycardia
Pain at the administration site
Pain
Dizziness
Contusion
Oropharyngeal pain
Skin abrasion
Seasonal allergy
fracture
Frequency undefined:
hypersensitivity
Contraindications
None
Caution
Hypersensitivity reactions
Pregnancy consideration: Insufficient data available
Lactation: Insufficient data available
Pregnancy category:
Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.
<b>Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.
Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.
Category D: adequate data available with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.
Category N: There is no data available for the drug under this category
Pregnancy consideration: Insufficient data available
Lactation: Insufficient data available
Pharmacology
It is a PMO used to treat duchenne muscular dystropy caused by genetic mutations. It works by binding to dystrophin gene sequence and dystrophin protein production.
Pharmacodynamics
Golodirsen is a oligonucleotide that skips the exon 53 in the processing of RNA and produces a dystrophin protein in muscle cells
Pharmacokinetics
Absorption: The drug gets absorbed through intravenous infusion.
Distribution: The protein binding range is 33-39%. The volume of distribution (Vd) is 668 mL/kg.
Metabolism: It does not metabolize and excrete unchanged in the body.
Elimination and Excretion: The elimination half-life is 3.4 hours. Plasma clearance is 346 mL/hr/kg. The majority of drug is eliminated unchanged in urine.
Administration
The IV solution involves a preservative-free solution that should be diluted before use. The solution must be clear and colorless. The dose should be calculated and diluted in 0.9% NaCl solution and infusion is given within 4 hours. It is stored at 2 to 8°C for one day.
Patient Information Leaflet
Generic Name: golodirsen
Pronounced: [ GOE-loe-DIR-sen ]
Why do we use golodirsen?
It is used in the treatment of mutations in Duchenne muscular dystrophy.
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