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DOI: 10.1055/s-0039-1698256
Ataluren in Patients Aged 2 to < 5 Years with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD): 28-week Results from a Phase 2 Study
Publication History
Publication Date:
11 September 2019 (online)
Introduction: Nonsense mutation Duchenne muscular dystrophy (nmDMD) is a rare X-linked disorder that results in a progressive decline in muscle function and early death. Ataluren is conditionally approved by the EMA for use in ambulant nmDMD patients ≥ 2 years. Initiation of ataluren for dystrophin restoration at a younger age may maximize therapeutic benefit.
Objective: Evaluate safety and pharmacokinetics of ataluren in male nmDMD patients ≥2 to <5 years.
Methods: This was an observational, open-label Phase 2 study that evaluated the safety and pharmacokinetics of ataluren (10, 10, and 20 mg/kg) in male nmDMD patients ≥2 to <5 years (n = 14). Changes in TFTs and the 3- and 8-part NSAA were assessed.
Results: Half of patients reported an adverse event, all were unrelated to ataluren. Improvements were noted in TFTs, and NSAA scores.
Conclusions: The safety profile in this younger population of patients with nmDMD was similar to the known safety profile of ataluren.
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No conflict of interest has been declared by the author(s).