Expanded Access Policy

Expanded Access Policy
 
Spark Therapeutics focuses on discovering and developing novel gene therapy products for genetic diseases. The following is Spark Therapeutics’ global expanded access policy for investigational therapies that are intended to treat serious diseases:
 
1.     Contact Information. At this time, Spark Therapeutics does not have any investigational gene therapy product candidates available for expanded access. If you have questions about our investigational product candidates or expanded access, please submit them to medinfo@sparktx.com.
 
2.     Request Procedures. Spark Therapeutics is not currently making its investigational gene therapy product candidates available on an expanded access basis anywhere in the world. Requests for expanded access to Spark Therapeutics’ investigational gene therapy product candidates must come from the patient’s treating physician because only physicians can enroll patients in a clinical trial or expanded access program (EAP).

General Criteria. Spark Therapeutics is not currently making its investigational gene therapy product candidates available on an expanded access basis anywhere in the world. In the event Spark Therapeutics decides to consider making one or more of its investigational gene therapy product candidates available through an EAP, Spark Therapeutics will evaluate and respond to each expanded access request that it receives on a case-by-case basis using criteria that ensures such requests are considered in a fair and consistent manner.
 
3.     Anticipated Timing. Spark Therapeutics anticipates that it will acknowledge receipt of any expanded access questions or requests within seven business days of receipt.
 
4.     Clinicaltrials.gov. As stated above, Spark Therapeutics is not currently making its investigational gene therapy product candidates available for expanded access use anywhere in the world. In the event that Spark Therapeutics decides to make its investigational product candidates available on an expanded access basis, this policy will be updated with a hyperlink to the expanded access record on clinicaltrials.gov after such record becomes active.
 
The posting of this policy by Spark Therapeutics shall not serve as a guarantee of access to any specific investigational drug by any individual patient. As authorized by and in accordance with the 21st Century Cures Act, Spark Therapeutics may revise this posted expanded access policy at any time.