Cook MyoSite - Expanded Access Policy

Cook MyoSite, Inc. (Cook MyoSite) strives to make regenerative medicine part of everyday medicine. Cellular therapies, a branch of regenerative medicine, introduce new cells to a region of the body to help restore or strengthen damaged or weakened tissue. Currently, Cook MyoSite is conducting clinical trials that study an autologous (cells or tissues from the same person) cell therapy to potentially repair muscle tissue.

Cook MyoSite is committed to conducting rigorous, controlled clinical trials with variable inclusion and exclusion criteria based upon the indicated use under investigation. These trials are designed to demonstrate to regulatory authorities that our investigational product is safe and effective, and as a result, allow our investigational cell therapy product to become a valuable treatment option for a wide range of patients. Participation in our clinical trials is the first and most preferable route to access our investigational product.

We understand that there may be patients with a serious, life-threatening disease or condition that have explored all other treatment options and are unable or ineligible to participate in our clinical trials. Expanded access, otherwise known as “compassionate use” or “pre-approval access,” is a potential pathway to access an investigational product that a patient may otherwise be unable to receive.

Cook MyoSite will consider providing an investigational product to a qualified requesting physician currently licensed within the United States via the United States Food and Drug Administration’s (FDA’s) expanded access pathway outside of an active clinical trial when the following general requirements are met:

• The patient has a serious or life-threatening disease or condition with no satisfactory alternative;
• Positive assessment that the anticipated benefits outweigh the risks to the patient;
• Positive assessment that Cook MyoSite has an adequate supply of resources for producing the investigational product;
• A determination that expanded access will not interfere with Cook MyoSite’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients;
• The patient can undergo a muscle biopsy procedure(s), and;
• The patient can undergo Cook MyoSite’s required donor screening and testing.

All requests for expanded access to our investigational product must be made by a patient’s treating physician. The treating physician must agree in writing to obtain necessary ethical and regulatory clearances, including informed consent, and to fulfill all regulatory reporting requirements concerning treatment outcomes. The treating physician must also agree to meet any applicable legal and regulatory requirements related to providing expanded access to an investigational product, as well as any Cook MyoSite requirements in terms of medical criteria, safety reporting, and protection of intellectual property. Each patient and their circumstances are unique, and as such, requests will be evaluated on a case-by-case basis in a fair and equitable manner.

Treating physicians may submit requests for expanded access to Cook MyoSite’s investigational product by email to MYO-ExpandedAccess@CookMyoSite.com. Each request submitted to this monitored inbox will be acknowledged with a reply from the company within 5 business days of receipt. All decisions to grant or deny expanded access to Cook MyoSite’s investigational product are final.