Expanded Access Policy

Chondrial Therapeutics, Inc. is an emerging biotechnology company focused on the treatment of rare diseases. Chondrial is currently developing CTI-1601, an investigational drug intended for the treatment of Friedreich’s ataxia (FRDA). A crucial step in the development of an investigational drug is to conduct clinical trials. Drugs being studied in clinical trials have unknown benefits and unknown risks that will not be understood until the clinical trials are complete.

 

Eventually, if results from the clinical trials are favorable, they will be submitted to FDA and other regulatory bodies for review of the drug’s safety and efficacy in order to seek approval for the drug. Obtaining regulatory approval for a new medicine is the best way to bring rapid access to the greatest number of patients who may benefit.

 

Sometimes patients may be able to access investigational drugs outside of a clinical trial. In the United States, this is possible through “expanded access,” sometimes also referred to as “compassionate use.” Unlike the use of an investigational drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for treatment purposes, rather than to gather data on safety and effectiveness.

 

In the early stage of drug development it is critical to systematically obtain information about the safety and tolerability of the investigational drug in a controlled manner. It is also important to evaluate investigational drugs under controlled conditions to assess their efficacy. Data on safety, tolerability and effectiveness is not yet available for CTI-1601. Therefore, Chondrial is not accepting expanded access requests at this time. We will reevaluate this policy in the future as information from our clinical trials become available. If the policy changes, this webpage will be updated.

 

If you have questions about this policy, or would like information about how to enroll in our clinical studies, please contact us at: https://chondrialtherapeutics.com/contact-us/. You can also obtain information about the study at https://clinicaltrials.gov/.

 

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