information: Presentation of results at a congress
Announcement: presentation of results at the 23rd International Annual Congress of the World Muscle Society
Company: PTC Therapeutics (USA - NJ)
Product: Translarna™ (ataluren)
- protein restauration therapy/protein restauration therapy/inducer of ribosomal readthrough on nonsense mutation mRNA stop codons. Ataluren (3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid) is an investigational new drug designed to enable the production of functional dystrophin protein in the muscle cells of patients with genetic disorders due to a nonsense mutation. Ataluren is designed to allow the ribosome to ignore the premature stop signal and continue translation of the mRNA, resulting in formation of a functioning protein. Ataluren does not cause the ribosome to read through the normal stop signal.
Disease: Duchenne muscular dystrophy
area: Rare diseases - Genetic diseases - Neuromuscular diseases
- 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 9 July 2018, 216 patients with a mean age of 9.8 years had been enrolled across 11 countries in Europe and Israel.
- 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 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.
- • On October 6, 2018, PTC Therapeutics announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense mutation, when compared with published natural history. The data show that children and adolescents receiving Translarna in the real-world setting are continuing to walk years longer than untreated children, and are staying more physically able. A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age – up to 5 years later than seen with natural disease progression in untreated children. The data were presented as a late breaker at the 23rd International Annual Congress of the World Muscle Society in Argentina.
- Patients who received Translarna in routine clinical practice also experienced a slower decline in their physical function compared with the placebo arm of Phase 3 Study 020, as measured by a series of timed function tests. Safety outcomes for patients in the STRIDE Registry were consistent with the known safety profile of Translarna.
- The analysis was based on data captured from 216 patients, the majority of whom had not been previously enrolled in an ataluren clinical trial, across11 European countries and Israel. Patients had a mean age of 9.8 years at first assessment and had been diagnosed with Duchenne caused by a nonsense mutation at approximately five years of age. Almost 90% had previously or were still receiving corticosteroids.