At Spruce Biosciences, we are dedicated to developing first-in-class therapies that address serious, underserved neurological conditions. Our lead investigational product, tralesinidase alfa enzyme replacement therapy (TA-ERT), is being studied for the treatment of mucopolysaccharidosis Type IIIB (MPS IIIB; Sanfilippo Syndrome Type B).

We believe that participation in a clinical trial is the most appropriate path to access investigational therapies while ensuring safety, consistent data collection, and regulatory compliance. Our focus remains on completing all necessary studies to support potential regulatory approvals as efficiently and safely as possible.

We understand that patients and their families may seek access to investigational medicines outside of clinical trials. Expanded Access, also known as Compassionate Use, is one potential pathway to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Consistent with guidance published by the U.S. Food and Drug Administration, we consider the following criteria to determine if an investigational medicine can be made available for treatment outside of a clinical trial:

• The individual has a serious or immediately life-threatening disease or condition;
• There is no comparable or satisfactory alternative therapy for the disease or condition;
• The individual does not qualify to participate in any ongoing clinical trial;
• Sufficient clinical evidence of safety and effectiveness for the indication has been established and concluded that the potential benefit outweighs the potential risks;
• The investigational medicine is under active development for the requested disease indication;
• Making the investigational medicine available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access;
• Adequate supply of investigational medicine exists; and
• There is a regulatory mechanism in the country or region to support expanded access.

Investigational medicines that have not yet received regulatory approval have not established their potential risks and benefits. Physicians and patients should consider all possible benefits and risks when seeking expanded access to an investigational medicine. We encourage individuals to speak directly with their treating physician regarding current or future options, clinical trials, and expanded access programs. 

Currently, we do not have any investigational products available for Expanded Access.

We are committed to advancing therapies that have the potential to improve the lives of individuals with serious or immediately life-threatening conditions and appreciate the trust placed in our work by the patient and medical communities.

In accordance with the 21st Century Cures Act, we may revise this policy at any time.