PTC Therapeutics, Inc. (PTC) is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. While our discovery programs are directed at targets in multiple therapeutic areas, we are focusing particularly on the development and commercialization of treatments for orphan and ultra-orphan disorders.
Our lead product candidate is ataluren for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) and nonsense mutation cystic fibrosis (nmCF). Ataluren has been approved under the trade name Translarna™ in the European Union for ambulatory patients with nmDMD who are 5 years and older.