Long-Term Benefit of Ataluren Treatment in Non-Sense Duchenne Muscular Dystrophy Versus Standard of Care

OBJECTIVES:

To present data on real-world effects of ataluren when compared with standard of care.

METHODS:

A EMBASE literature search was performed using the terms [ataluren] and [standard of care].

RESULTS:

The search brought 48 results and 3 relevant and most recent studies were selected.

Data from a long-term phase 3 study of patients with DMD treated with ataluren were compared with those from patients in a natural history study treated with standard of care (CINRG DHNS). Ataluren + SoC was associated with a 2.2-year delay in age at LoA, and a 3.0-year delay in decline of predicted forced vital capacity to<60% in non-ambulatory patients, versus SoC. Ataluren + SoC was also associated with a delay of approximately 1 year in the age at predicted FVC declining to <50%, compared with SoC alone. In addition, 1 patient (2.2%) from Study 019 and 9 patients (20.0%) from the CINRG DNHS experienced a FVC decline below the critical threshold of < 1 L.

STRIDE is a registry that provides ataluren real-world data in patients with nmDMD. Two studies compared the data from the STRIDE with the CINRG DNHS. It was observed that ataluren + SoC delayed age at LoA in 5.4 years compared to CINRG (SoC only). Ataluren + SoC was also associated with a delay of approximately 1.8 year in the age at predicted FVC declining to <60%, compared with SoC alone.

CONCLUSIONS:

These data suggest ataluren + SoC slow disease progression through age at loss of ambulation and pulmonary function.