PTC Therapeutics Inc (PTC Therapeutics) is a biopharmaceutical company that discovers, develops and commercializes medicines for rare genetic disorders. The company classifies its operations in one operating segment. The marketed products of PTC Therapeutics include Translarna (ataluren), Emflaza (deflazacort), indicated for the treatment of Duchenne muscular dystrophy, and Evrysdi (risdiplam) for the treatment of pediatric and adult patients with spinal muscular atrophy.

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PTC Therapeutics focuses on advancing its pipeline candidates including PTC596 for the treatment of leiomyosarcoma; PTC299 for the treatment of acute myelogenous leukemia; PCT518 for the treatment of Huntington’s disease; PTC857 for children with mitochondrial disease; PTC923 for phenylketonuria and PTC-AADC for the treatment of aromatic L-amino acid decarboxylase deficiency. Its R&D expenses include the clinical development costs associated with its product development programs and R&D costs associated with its discovery programs. In FY2022, PTC Therapeutics incurred R&D expenses of US$651.5 million representing an increase of 20.5% in FY2022. The company has 143 active U.S. patents and 63 pending U.S. non-provisional patent applications, 42 issued in U.S. patents. It has patent rights relating to other jurisdictions including Canada, certain South American countries, Europe, certain Asian countries, certain Middle Eastern countries, certain African countries, and certain Eurasian countries