golodirsen

Action and Spectrum

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.  

Drug Interaction

Adverse Reaction

Frequency defined:   

>10%  

Pyrexia  

Abdominal pain  

Cough  

Nausea  

Headache  

Fall  

Nasopharyngitis  

Vomiting  

1-10%  

Back pain  

Diarrhea  

Ligament sprain  

Influenza  

Rhinitis  

Ear infection  

Tachycardia  

Constipation  

Pain at the administration site  

Pain  

Dizziness  

Contusion  

Oropharyngeal pain  

Skin abrasion  

Seasonal allergy  

fracture  

Frequency undefined:   

hypersensitivity 

Black Box Warning

Contraindication / Caution

Contraindications  

None  

Caution  

Hypersensitivity reactions 

Pregnancy / Lactation

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 

Pharmacology

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. 

Adminstartion

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

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.