Translarna delays ambulation loss in boys with Duchenne muscular dystrophy
Click Here to Manage Email Alerts
PTC Therapeutics Inc. released results showing that treatment with Translarna delayed loss of ambulation by more than 5 years among boys with nonsense mutation Duchenne muscular dystrophy vs. standard of care alone.
The results were part of the Strategic Targeting of Registries and International Database of Excellence (STRIDE) patient registry, an ongoing, multicenter, observational study of the safety and effectiveness of Translarna (ataluren, PTC Therapeutics) in routine care.
Study participants treated with ataluren and standard of care exhibited a delay of 1.8 years in pulmonary function decline.
"The 5-year analysis of the STRIDE registry clearly demonstrates Translarna’s profound impact on changing the course of disease progression,” Stuart W. Peltz, PhD, CEO of PTC Therapeutics, said in a press release. “We are all proud to see Translarna’s life-changing effect on boys with Duchenne. The results’ robustness showing both a 5-year delay in loss of walking, as well as its ability to prolong lung function, confirms what we have seen in our clinical trials.”
Results of a time-to-event analysis of 5 years of registry data from 241 boys in 13 countries showed that boys treated with ataluren plus standard of care had a median age of loss of ambulation of 17.9 years vs. 12.5 years for those on standard of care alone. A total of 80% of boys who received ataluren plus standard of care were still walking at age 12 years compared with 52% who received standard of care alone. Boys treated with ataluren reached a predicted forced vital capacity lower than 60% at a median age of 17.6 years vs. 15.8 years for those who did not receive ataluren.
According to the release, the loss of lung function delay was critical, because the sub-60% threshold represents the point at which patients typically begin to require respiratory physical therapy.
Ataluren was well tolerated among this patient population. Safety results represented 1,059 patient-years of exposure and remained consistent with the known safety profile of the therapy.
“These results add to the totality of the evidence of Translarna’s benefit for the patients and their families,” Peltz said in the release.