"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,
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.
About Translarna (ataluren)
Translarna (ataluren), discovered and developed by
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. Enrolled patients will be followed for at least 5 years from the date of enrollment, or until withdrawal from the study. As of
Effectiveness information may include neuromuscular function (as measured for example by timed-function tests, the North Star Ambulatory Assessment, and Performance of the Upper Limb (PUL) measures, cardiac function (including echocardiogram where available), pulmonary function (including spirometry measures), 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
The Registry also fulfils a post-marketing commitment to the
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
Further information about TREAT-NMD can be found here: http://www.treat-nmd.eu/
The CINRG DNHS enrolled patients aged 2–28 years with DMD at 20 centers in nine countries from 2006 to 2016. Data from CINRG DNHS patients receiving standard of care (SoC) are used in the present analysis as a control to provide context for assessing the effects of ataluren plus SoC in patients in the STRIDE Registry. SoC refers to palliative therapies and corticosteroid treatment.
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties.
More information regarding Duchenne is available through the
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us on www.ptcbio.com and follow us on
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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.
2. Mayer OH et al - Characterization of Pulmonary Function in Duchenne Muscular Dystrophy. Pulmonol. 2015;50:487-494.
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; expansion of commercialization of Translarna and related regulatory submissions; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to complete a dystrophin study necessary to support a re-submission of its Translarna NDA for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) to the
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
[*] STRIDE: Strategic Targeting of Registries and International Database of Excellence
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