PTC Therapeutics announces conditional EU approval of Translarna PTC Therapeutics announced that the European Commission has granted conditional marketing authorization for Translarna, in the European Union for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. The authorization allows PTC to market Translarna in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway. As part of the conditional marketing authorization, PTC is obligated to complete its confirmatory Phase 3 trial in nmDMD and submit additional efficacy and safety data from the trial.
NHS to wait on NICE to make call on PTC Therapeutics, BioMarin drugs NHS England has set out its planned investment decisions for certain specialized services as part of its annual commissioning round. NHS England has agreed with the recommendations from CPAG that final funding decisions on the following two treatments should be made after NICE has concluded its Highly Specialized Technology Appraisal process: BioMarin Pharmaceutical's (BMRN) Vimizim for Morquio A Syndrome and PTC Therapeutics' (PTCT) Translarna for Duchenne muscular dystrophy. The decisions on those two drugs came among 39 proposed new investments that the group said were "carefully considered against the principles NHS England follows when making investment decisions." Reference Link