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 due to a nonsense mutation and for certain cancers, but does not yet have any approved drugs. 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:

  • Whether the disease or condition is serious or life-threatening.
  • If the request is for a medication that is specific to a particular mutation, whether there is adequate documentation that the patient has genotyping that is consistent with that mutation.
  • Whether the indication for which the medication is requested is consistent with and not likely to interfere with PTC’s overall development plan for the medication.
  • Whether there are adequate alternative therapies or clinical trials available.
  • Whether there is substantial scientific evidence to support both the safety and the efficacy of a product for the indication requested and sufficient clinical data are available to identify an appropriate dose.
  • Whether the potential benefit to the patient justifies the potential risks of the treatment with the investigational medicine and those potential risks are not unreasonable in the context of the disease or condition to be treated.
  • Whether allowing expanded access or compassionate use will interfere with the initiation, conduct, or completion of clinical trials that could support marketing approval of the investigational medicine for the disease or condition for which expanded access or compassionate use is sought.
  • Whether adequate and appropriate drug supply is available.
  • The qualifications of the physician, including experience with the therapy or expertise with the specific disease or condition and ability to administer and monitor the patient’s safety.
  • Whether the potential patient and/or their guardian is willing and able to provide informed consent and comply with the safety and monitoring requirements defined by PTC

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.

For further information on available clinical trials and expanded access programs, visit and search for trials where “PTC” is the sponsor.

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