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Real-World Data Shows Ataluren Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy

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PTC Therapeutics, Inc today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Pulmonary function was also preserved in those treated with Translarna.1 The analysis, presented in the publication of an interim analysis of preliminary real-world data, compared children treated with Translarna in a real-world setting from the STRIDE[*] registry with a matched cohort in a long-term natural history study, CINRG[†].1 In addition, no new safety signals were observed in the patients treated with Translarna, consistent with what has been shown in previous clinical trials. The interim data have been published in the Journal for Comparative Effectiveness Research. Final data from the STRIDE registry is expected in 2025.

“Duchenne muscular dystrophy is a devastating disease that causes irreversible muscle wasting and progressively robs young people of their ability to walk, move, and breathe naturally without a ventilator, and it reduces their autonomy in daily life tasks,” said Dr. Andrés Nascimento, Pediatric Neurology, Neuromuscular Diseases Unit, SJD Children’s Hospital, Barcelona, Spain. “In a real-world setting, children and adolescents treated with Translarna experience a delay in the disease progression, are able to maintain more mobility, and have a higher level of physical autonomy concerning the course of the natural history of the disease. This is not only clinically relevant, but especially important for the quality of life of patients and their families.”

Children treated with Translarna in a real-world setting as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort in the CINRG natural history study, with a median age at loss of ambulation of 14.5 years and 11 years, respectively (72% relative risk reduction). 1

Additional analyses from the registry demonstrated that Translarna sustained the ability of boys with Duchenne to complete everyday tasks by years compared with the natural history cohort. 1 In timed function tests, Translarna sustained their ability to stand up from lying down, in under 5 and 10 seconds, for three years longer than in boys treated with SoC alone.1 Boys treated with Translarna were also still able to climb four stairs in under 5 and 10 seconds for 1.5 and 3.6 years longer, respectively, than boys on SoC alone. 1

In addition, the analysis showed a trend toward delayed worsening of pulmonary function in routine clinical practice for patients treated with Translarna, compared to the matched patients in CINRG. Researchers evaluated FVC, a traditional measure of lung function in Duchenne patients that correlates with disease progression and mortality. 1,2 The STRIDE data showed that 32.1% of standard of care patients from the natural history cohort had an FVC of <50%, compared to only 2.2% of patients receiving Translarna.1,2 However, the authors state that given the low number of events and the shorter duration of follow-up of patients in the STRIDE registry compared to CINRG in these interim analyses, it is premature to draw firm conclusions from these results. 1 After loss of ambulation and loss of the use of the arms, the respiratory muscles of people with Duchenne start to progressively deteriorate, leading to the risk of life-threatening respiratory complications and the need for ventilation support. 1

“The data from the STRIDE registry are consistently confirming the benefits seen in Translarna clinical trials and the difference it is making to patients and their families – more years of being independent and physically able without reliance on a wheelchair or ventilator,” said Dr. Claudio Santos, SVP, Global Medical Affairs, PTC Therapeutics.

Read the full press release, here.

References

1.  Mercurio E, Muntoni F and Nascimento O et al. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. Journal for Comparative Effectiveness Research. 2020. Available at: 

2.  Mayer OH et al – Characterization of Pulmonary Function in Duchenne Muscular Dystrophy. Pulmonol. 2015;50:487-494.

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