PTC Therapeutics: EMA adopted negative opinion on MAA for Atuluren approval PTC Therapeutics announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a negative opinion on the company's marketing authorization application for conditional approval of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy. The response from the CHMP is consistent with the company's previous guidance concerning the substantial risks regarding conditional EMA approval and underscores the importance of PTC's work in completing the ongoing confirmatory Phase 3 clinical trial. In April 2013, PTC initiated a 48-week, 220-patient confirmatory Phase 3 clinical trial of ataluren for the treatment of nmDMD. The trial is on track to complete enrollment in mid-2014 with top-line data expected in mid-2015. PTC intends to request a re-examination of the CHMP opinion with a final outcome expected in the second quarter of 2014, when the confirmatory study is expected to be more fully enrolled.
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