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MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › Other drugs for disorders of the musculo-skeletal system
ataluren
M09AX03
Forms & Strengths
- Granules for oral suspension: 125 mg packets
- Granules for oral suspension: 250 mg packets
- Granules for oral suspension: 1000 mg packets
Adult Dosing
- Not applicable; indicated for pediatric patients with nonsense mutation Duchenne muscular dystrophy
Pediatric Dosing
- Duchenne muscular dystrophy (patients aged 2 years and older): 10 mg/kg dose in the morning, 10 mg/kg in the midday, and 20 mg/kg in the evening (total daily dose of 40 mg/kg) administered orally
Indications
- Treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older
Mechanism of Action
- Protein translation readthrough drug designed to enable the formation of a full-length, functional dystrophin protein by bypassing premature stop codons (nonsense mutations) in the mRNA
Contraindications
- Hypersensitivity to ataluren or any of the excipients
- Concurrent administration of potent IV nephrotoxic agents
Adverse Reactions
- Vomiting
- Diarrhea
- Nausea
- Abdominal pain
- Headache
- Cough
- Nasopharyngitis
- Blood triglyceride elevations
- Blood pressure increased
Drug Interactions
- OAT1/3 substrates (e.g., oseltamivir, acyclovir): Ataluren may inhibit OAT1/3, potentially increasing plasma concentrations of these substrates
- UGT1A9 substrates: Potential for altered clearance
- BCRP and OATP1B1/3 substrates: Co-administration may affect transporter-mediated disposition
Curated Content: Needs Vetting Before Put to Clinical Use