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.

* EVRYSDI is a product from the SMA collaboration between PTC, the SMA Foundation and Roche. EVRYSDI is marketed in the United States by Genentech, a member of the Roche Group, and marketed in rest of world by Roche.

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.

U.S. Expanded Access Policy

Our Science

Leveraging our expertise and cutting-edge biotechnology platforms, we’re urgently turning the latest scientific advances into purposeful treatments

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Therapeutic Areas

Diseases we’re on a mission to change

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Clinical Trials

We're committed to making progress in rare disease through clinical trial research, each and every moment of every day

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