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U.S. Expanded Access Policy

At PTC Therapeutics, Inc. (“PTC”), we are committed to providing novel treatment options to patients living with serious diseases. To do this, PTC conducts 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.

PTC maintains that participating in clinical trials is the best way for patients to access medicines prior to approval. In some circumstances when a clinical trial is not possible, patients with serious diseases or conditions may seek special access to investigational medicines. This access to investigational medicines is often referred to as expanded access or compassionate use. In the United States, PTC is engaging in development of drugs for genetic disorders. Because of the need to focus development efforts on clinical trials that could support regulatory approval, PTC generally will not provide access to its investigational medicines via expanded access or compassionate use. However, PTC is willing to evaluate requests for such access in the United States from qualified physicians, including consideration of the following:

All questions and requests regarding PTC’s expanded access or compassionate use programs should be submitted to PTC by email: In the case of requests by treating physicians, the email should contain information sufficient for PTC to evaluate the request consistent with this policy. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements defined by PTC. Requests for expanded access or compassionate use may only be made by licensed US physicians. PTC will acknowledge receipt of requests within 5 business days. PTC cannot guarantee that the investigational medicine will be available for a particular patient under an expanded access or compassionate use program. If granted, access will be for a designated period of time and then a request must be submitted and reviewed to continue the program.

At any point, expanded access or compassionate use can cease being made available at the discretion of PTC. This policy may change without notice.