PTC THERAPEUTICS, INC.

We are a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. We are advancing a robust and diversified pipeline of transformative medicines as part of our mission to provide access to best-in-class treatments for patients with unmet medical needs. Our strategy is to leverage our scientific expertise and global commercial infrastructure to optimize value for our patients and other stakeholders. Our Pipeline We have a diversified therapeutic portfolio that includes several commercial products and product candidates in various stages of development, including discovery, research and clinical stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. The disclosure below summarizes the status of our significant commercial products and clinical-stage programs as of the date of this report, including those with our strategic partners: Global Commercial Footprint o Sephience TM (sepiapterin) Sephience is our product for the treatment of adult and pediatric patients with phenylketonuria, or PKU, a rare, inherited metabolic disease, characterized by the body's inability to break down an essential amino acid called phenylalanine, or Phe, which can result in neurological and other symptoms. Through its novel dual mechanism of action, Sephience is able to effectively reduce blood Phe levels and has the potential to treat a broad range of PKU patients.