PTC Therapeutics, Inc.

PTC Therapeutics, Inc. is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare diseases. The company's mission is to provide access to best-in-class treatments for patients with high unmet medical needs, focusing on addressing the underlying genetic causes of disease. Its corporate headquarters are located in Warren, New Jersey, U.S.

PTC Therapeutics' portfolio includes approved therapies such as Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, Emflaza (deflazacort) also for Duchenne muscular dystrophy, and Upstaza (gene therapy, also known as Kebilidi in the U.S.) for aromatic L-amino acid decarboxylase (AADC) deficiency. They also have commercialization rights for Tegsedi (inotersen) and Waylivra (volanesorsen) in Latin America and certain Caribbean countries, and Evrysdi (risdiplam) for spinal muscular atrophy (SMA) through a collaboration with Roche. The company's pipeline includes investigational therapies like Sepiapterin for phenylketonuria (PKU) and PTC518 for Huntington's disease.

In recent news, PTC Therapeutics reported positive topline results from the 24-month interim analysis of the PIVOT-HD extension study of Votoplam in April 2026. The company also announced the FDA approval of Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in children and adults. PTC Therapeutics has been recognized as a 2026 Gallup Exceptional Workplace Award winner for the fifth time, reflecting its culture and commitment to employee development. Dr. Matthew B. Klein serves as the Director and Chief Executive Officer. The company continues to invest in a diverse pipeline for conditions with significant unmet needs, leveraging its scientific platforms in splicing, inflammation, and ferroptosis.