A pipeline of innovation and hope
Approved Medicines
PTC has been a pioneer in the development of breakthrough rare disease therapies, delivering a number of firsts for patients who previously had no other treatment options. Our strategy leverages our innovative scientific knowledge, our deep therapeutic expertise and robust global commercial infrastructure. See below for a list of our approved/authorized products. Please note, our products are approved in individual countries for specific uses and the information provided is governed by local regulations. The name, availability and indication of these medicines may vary by country.
U.S. Expanded Access Policy
At PTC we are committed to providing novel treatment options to patients living with serious diseases. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, which may allow us to obtain the necessary regulatory approvals and provide patients with broader access to these medicines.
Leveraging our expertise and cutting-edge biotechnology platforms, we’re urgently turning the latest scientific advances into purposeful treatments
We're committed to making progress in rare disease through clinical trial research, each and every moment of every day