Expanded Access Policy

Alkeus Pharmaceuticals, Inc. is dedicated to enhancing patient outcomes for those affected by severe retinal disorders. Our leading investigational therapy, gildeuretinol acetate (ALK-001), aims to address the unmet needs of patients living with Stargardt disease.

In alignment with this commitment, Alkeus in collaboration with our partner clinical sites, conducts clinical trials to ascertain the safety and efficacy of our investigational therapy. We believe that participating in a clinical trial is the optimal way for patients and healthcare providers to access investigational treatments before they receive regulatory approval. In such trials, patients benefit from direct physician supervision, thorough monitoring and comprehensive follow-up, ensuring the safe administration of these investigational therapies. Therefore, Alkeus strives to direct individuals seeking access to our investigational treatments to suitable clinical trials whenever possible. For more information, please visit the “Science” page on our website at or visit

Alkeus recognizes that some patients face serious and immediately life-threatening conditions for which no satisfactory treatment options exist, and may not be able to participate in a clinical trial. Expanded access, also known as compassionate use, involves providing an investigational product that has not yet been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority outside the U.S.

Providing an investigational product though an expanded access program is a complex and resource-intensive process. Alkeus is hopeful to provide expanded access for eligible patients in the future. We will regularly reassess our capability to facilitate expanded access and will publicly disclose our related policy should an expanded access program become available through Alkeus.

For additional information on expanded access programs, please search “expanded access programs” at

Contact Information for Expanded Access Requests