PTC Therapeutics announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, has recommended approval of expanding the indication of Translarna to include ambulatory children aged two to five years with nonsense mutation Duchenne muscular dystrophy, or nmDMD. This broadens the use beyond the current indication which is for ambulatory patients who are over five years of age. This recommendation is based on the CHMP’s review of PTC’s Study 030 trial results which the CHMP concluded demonstrates a positive benefit-risk ratio in this population. In addition to the label expansion, the CHMP has also recommended the renewal of the current marketing authorization of Translarna. PTC’s focus on early patient identification and market readiness have been intensified in anticipation of the CHMP recommendation and the launch of Translarna for patients as young as 2 years of age is planned to start immediately at the time of EC ratification.
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