Expanded Access Policy

Expanded access, also called compassionate use, refers to the use of an investigational therapy outside of a clinical trial when the primary purpose is to diagnose, prevent or treat a serious or immediately life-threatening condition in a patient.

Importantly, clinical trials are conducted to determine if an investigational therapy would be safe enough and effective for patients to use. Given the early stage of our clinical development programs, the safety and efficacy of our treatments is still being assessed. Therefore, we believe that participation in one of our clinical trials is the most appropriate way to access our investigational therapies and we are not currently making our investigational therapies available through an expanded access program or for compassionate use.

We considered several factors, consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines when determining our current policy. They include:

The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or open clinical trials that are applicable)

There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information

The ability to provide a therapy in a fair and equitable manner, so that there is adequate manufacturing capacity to provide therapies across all ongoing clinical trials and expanded access programs

Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients

We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy. Hemab is committed to reviewing our Expanded Access Policy and may revise this policy as we better define the safety and efficacy of our treatments.

If you have additional questions, please speak with your physician or contact clinicaltrials@hemab.com. Hemab regularly monitors this mailbox and will use our best efforts to acknowledge each submitted requested within three business days after receipt.