PTC Therapeutics Announces First Publication of Real-World Data Showing Translarna™ (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy
- STRIDE registry analysis shows Translarna preserved ambulation and physical function by years compared with those in CINRG Duchenne Natural History Study, with no new safety signals
- The trend toward delayed worsening of pulmonary function compared with natural history study
- Read the full press release here
PTC Therapeutics, Inc. 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. 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. 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. The final data from the STRIDE registry is expected in 2025.
Dr. Andrés Nascimento, Pediatric Neurology, Neuromuscular Diseases Unit, SJD Children’s Hospital, Barcelona, Spain – “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. 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.
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. 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. 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.
Also, 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. After the 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.
Dr. Claudio Santos, SVP, Global Medical Affairs, PTC Therapeutics – “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.”
About Translarna (ataluren)
Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older. Ataluren is an investigational new drug in the United States. In the video, Dr. Ellen Welch answers our questions about nonsense mutation and ataluren > Here
Ataluren in Canada
At this moment, PTC Therapeutics has not filed an application for marketing approval with Health Canada, but it has started a conversation with the legislator.
About the STRIDE Registry
The STRIDE (Strategic Targeting of Registries and International Database of Excellence) Registry is an ongoing, multicenter, observational study of the safety and effectiveness of Translarna in routine care. It is the first patient data repository to provide real-world experience regarding the long-term use of Translarna in routine clinical practice.
Effectiveness information may include neuromuscular function measures, cardiac function, pulmonary function, and quality of life measures. Assessments of musculoskeletal health, rehabilitation, orthopedic and gastrointestinal management, as well as other measures of psychosocial management, will be collected to allow for comparison of patient health-management activities in routine clinical care to those of published treatment guidelines.
STRIDE is a collaborative partnership between TREAT-NMD and PTC Therapeutics, led by a Steering Committee comprised of leading experts in Duchenne, patient advocates from around the world and PTC representatives.
The Registry also fulfils a post-marketing commitment to the Pharmacovigilance Risk Assessment Committee of the European Medicines Agency.
About TREAT-NMD
TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch, in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide. The network has developed from its European roots to become a global organization that brings together leading specialists, patient groups and industry representatives to ensure preparedness for the trials and therapies of the future while promoting best practice today.
Further information about TREAT-NMD can be found here > TREAT-NMD
About CINRG
The Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study was a prospective, longitudinal study of more than 400 patients with Duchenne muscular dystrophy (DMD) who were followed up between 2006 and 2016 at 20 worldwide centers as part of the academic clinical trial network, CINRG.
More links
- Read the full press release here
- Pioneer in DMD Therapy here
- Learn more about nonsense mutation here
- Learn more about ataluren (Translarna™) at ptcbio.com
SOURCE PTC Therapeutics, Inc.
