PTC Therapeutics, Inc.

PTC THERAPEUTICS, INC. is a global biopharmaceutical company focused on discovering, developing, and selling specialized medicines for children and adults with rare disorders. The company aims to provide access to treatments for patients with unmet medical needs by using its scientific knowledge and global sales network. PTC THERAPEUTICS has a diverse range of therapeutic products, including several that are already on the market and others in various stages of development, from early discovery to clinical trials. These efforts are concentrated on creating new treatments for rare neurological and metabolic diseases. As of the report date, the company's significant commercial products and clinical-stage programs include: Sephience (sepiapterin): This product treats adult and pediatric patients with phenylketonuria (PKU), a rare inherited metabolic disease. PKU prevents the body from breaking down phenylalanine (Phe), which can lead to neurological issues. Sephience works through a dual mechanism to reduce blood Phe levels and can potentially treat a wide range of PKU patients. Sephience is approved in the United States, the European Economic Area (EEA), Japan, and other regions. Global DMD Franchise: This franchise includes two products for Duchenne muscular dystrophy (DMD), a rare and life-threatening disorder: Translarna (ataluren) and Emflaza (deflazacort). Translarna previously held conditional approval in the EEA. However, in March 2025, the European Commission (EC) adopted a negative opinion from the Committee of Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), leading to the non-renewal of Translarna's conditional marketing authorization for treating nonsense mutation Duchenne muscular dystrophy (nmDMD).