EU conditional approval of PTC orphan drug translana for nonsense mutant dystrophy of Duchenne muscle (nmdmd)

Source: Biovalley PTC pharmaceutical announced on August 4, 2014 that the drug translanna (atauren) was approved by the European Commission (EC) conditionally for the treatment of ambulatory patients with 5-year-old and above nonsense mutant DMD This ratification is applicable to the 28 member states of the European Union and the member states of the European economic area, including Iceland, Liechtenstein and Norway As part of the conditional listing, PTC is obliged to complete the validation phase III act DMD study in nmdmd and submit additional efficacy and safety data from the study In January this year, the European Drug Administration (EMA) Committee on human pharmaceutical products (CHMP) gave trans larna a negative opinion, and then PTC company submitted additional analysis data and asked for re examination, after which CHMP proposed to approve trans larna conditionally in May this year Previously, the FDA has granted translana orphan status Translana's approval was based on data from a phase IIB study and subsequent analysis The study, a 48 week, placebo-controlled, double-blind trial, was conducted in 174 patients with nonsense variant Duchenne muscular dystrophy (nmdmd) The data showed that compared with the placebo group, the 6-minute walking distance (6MWD) in the treatment group increased by 31.3 meters on average, and the rate of falling off bed activity was slower Safety data showed that translana was generally well tolerated, and serious adverse events were rare At present, PTC company is carrying out phase III act DMD clinical research Translana is a protein repair drug developed for patients with genetic diseases caused by nonsense mutations It aims to make genes with nonsense mutations produce functional proteins If there is nonsense mutation in the gene, it will lead to the early termination of protein synthesis and produce non functional protein Currently, translana is being developed for the treatment of cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).