PTC Therapeutics Receives Positive Opinion from CHMP for Translarna™ (ataluren) - Seite 2
The CHMP opinion will form the basis for a European Commission (EC) decision as to whether to formally grant the conditional marketing authorization. The European Commission will review the positive opinion from the CHMP and generally delivers its final decision within three months. The conditional marketing authorization would authorize the company to market Translarna with unified labeling in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway.
"We congratulate PTC Therapeutics on this landmark decision by the CHMP," stated Elizabeth Vroom, Chair of United Parent Project Muscular Dystrophy (UPPMD). "We applaud PTC for its dedication to the community and its perseverance in pursuing regulatory options to provide Translarna to patients as rapidly as possible. The company's pioneering work has paved the way and encouraged the scientific community to develop new therapies for DMD," she continued. "The EMA is to be commended for recognizing the great unmet need for novel treatments for this relentlessly progressive disease."
Filippo Buccella, President of Parent Project Italy and UPPMD board member, commented, "After thirty years since the discovery of the dystrophin gene, we are finally beginning to see a change in the landscape. For the first time in the history of Duchenne, we see the path to approval for a drug to treat the underlying cause of DMD. This positive result rewards the efforts of a company that has always believed in collaboration with patients and with clinicians. The perseverance of this community made it possible to realize a dream that can give hope to the boys affected with nmDMD in Europe. Our work as a community of patients is not yet finished and we will stand ready to participate with PTC in the ensuing stages of this process and, we hope, with many other companies with new therapies for Duchenne."
PTC requested a re-examination of the CHMP's negative opinion received in January 2014. The positive opinion is based on data and subsequent analysis submitted from a 48-week, 174-patient Phase 2b double-blind, placebo controlled trial which demonstrated that nmDMD patients treated with Translarna (40 mg/kg given daily) walked on average 31.3 meters farther than patients on placebo, as measured by the change in six-minute walk distance (6MWD) from baseline to Week 48. Patients receiving Translarna also demonstrated a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD. Safety results showed that Translarna was generally well tolerated. Serious adverse events were infrequent and none were considered to be related to Translarna. PTC's global Phase 3 ACT DMD clinical trial is ongoing with full enrollment expected mid-2014.