Poxel

Patient Eligibility Criteria:
To be eligible for access to an investigational product, a patient must meet the following criteria:

• Be an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.
• Have undergone standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are not eligible or able to participate in any planned or ongoing clinical trial of the investigational product(s).
• Has manifestations of disease for which there is sufficient evidence of a projected benefit (e.g. based on prior positive clinical trial results) from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• Any other pertinent medical criteria for access to the investigational product, as established by Poxel.
• Investigational Product Criteria

In addition to the patient eligibility requirements, the investigational product must meet the following criteria:

• The product is in active clinical development.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product, and that there is sufficient investigational product available to ensure that supply constraints will not impede continued use of the product(s) by patient(s) or the conduct of our clinical trials.

Treating Physician Criteria and Responsibilities:
The physician(s) attending to the patient(s) who is/are receiving an investigational product through expanded use access is (are) properly licensed in the U.S. and fully qualified to administer the product. The physician must agree in writing to comply with:

• Any applicable legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any Poxel requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.