As PPMD reported in our World Muscle Society recap yesterday, PTC Therapeutics has announced that data from their STRIDE registry demonstrate that Translarna preserves lung function in children and adolescents compared with a matched cohort in a long-term natural history study. We are encouraged by this news and look forward to continued updates from PTC.
Read PTC Therapeutic’s Press Release
PTC Therapeutics Announces New Real-World Analysis Demonstrating Translarna™ (ataluren) Slows Disease Progression in Patients with Duchenne Muscular Dystrophy– Lung function data from the STRIDE Registry show a trend toward delay of decline of pulmonary function compared with those in CINRG Duchenne Natural History1 – SOUTH PLAINFIELD, N.J., Oct. 4, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that data from STRIDE,* the first international registry for patients with Duchenne muscular dystrophy due to a nonsense mutation receiving Translarnaä (ataluren), demonstrate that Translarna preserves lung function in children and adolescents compared with a matched cohort in a long-term natural history study.1 The real world analysis was presented at the 24th International Annual Congress of the World Muscle Society.”Across ambulation, physical function and lung function, the STRIDE data demonstrate that patients receiving Translarna preserved function for years longer than patients receiving standard of care,” said Stuart Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “These are real world results that provide clinicians and regulators the true picture of patient response to treatment.”
Researchers evaluated FVC, a traditional measure of lung function in Duchenne patients that correlates with disease progression and mortality.2 The STRIDE data showed that 32.1% of standard of care patients from the natural history cohort had a FVC of <50%, compared to only 2.2% of patients receiving Translarna after a mean total exposure of 633 days.1 The data also indicates that Translarna significantly preserved patients’ ability to stand up from lying and climbing stairs compared with natural history.3
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. Patients with a predicted FVC of <50% are considered to be in the late non-ambulatory stage of Duchenne.4 To conduct the analysis, patients from the STRIDE Registry were matched against a comparable cohort of patients from the Cooperative International Neuromuscular Research Group Natural History Study, based on their propensity for disease progression.1
These latest data build on the STRIDE registry – time-to-event analysis, which demonstrated that the median age at which patients on Translarna lost the ability to stand up from lying in under 5 seconds (the first key clinically significant Duchenne milestone) is 12 years – 3 years later than seen with natural disease progression in untreated children [9.1 years].3
“It’s very encouraging to see positive lung function results in a real world setting and provides reassurance that ataluren is slowing disease progression,” said Dr. Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University, and author of the study. “Respiratory failure is the primary cause of disability and death in patients with Duchenne and monitoring and preserving lung function is a key clinical priority. As the muscles progressively weaken, breathing and effective coughing gets more difficult and patients eventually become dependent on ventilation support, which is distressing for families.”
The demographic characteristics of the study population from the STRIDE Registry were published in August this year in the Journal for Comparative Effectiveness Research.5
* Strategic Targeting of Registries and International Database of Excellent (STRIDE)
1 Tulinius M et al. Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE Registry and the CINRG Natural History Study: A matched cohort analysis. Abstract presented at World Muscle Society 2019, 1-5 October, Copenhagen, Denmark.
2 Mayer OH et al – Characterization of Pulmonary Function in Duchenne Muscular Dystrophy Pulmonol. 2015;50:487-494. And Philips, M et al – Changes in Spirometry Over Time as a Prognostic Marker in Patients with Duchenne Muscular Dystrophy Am J Respir Crit Care Med Vol 164. pp 2191-2194, 2001
3 Mercuri, E., Buccella, F. et al. Timed function test data in patients with Duchenne muscular dystrophy from the STRIDE Registry and the CINRG Duchenne Natural History Study: a matched cohort analysis. Poster 11-38. Presented at EPNS 2019 (16-21 September, Athens, Greece).
4 Birnkrant DJ et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol 2018;17:347–61.
5 Muntoni, F., Desguerre, I. et al. Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry. J Comp Eff Res. 2019 Aug 15. doi: 10.2217/cer-2019-0086.