Alexion

Commitment to Patients

Alexion is committed to serving people affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Our goal is to bring safe and effective medicines to as many patients as possible by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities around the world. People living with certain rare diseases and devastating conditions often have few or no treatment options. Our work is marked by not only a commitment to the highest standards of preclinical and clinical research but also an acute sense of urgency. As such, we believe that the best way for patients to access our medicines in advance of commercial availability is by participating in a clinical trial. Alexion currently has approved and investigational medicines in clinical trials for a number of different diseases, which are listed in the Pipeline section of our website.

Criteria for Consideration

All requests received from a treating physician will be assessed on a case-by-case basis. Alexion cannot guarantee that requests for access will be granted. Each request will be considered based on whether certain conditions are met. These conditions include, but are not limited to:

• The patient must have a serious or immediately life-threatening condition
• The patient must have exhausted all other treatment options, including alternative approved medicines or clinical trials that are available
• There is sufficient evidence to demonstrate a favorable benefit-risk ratio
• Providing access will not jeopardize existing clinical trials or potential regulatory approval of the investigational medicine
• There is an adequate supply of the medicine for all ongoing clinical trials, as well as individual access requests
• Access can be provided in a fully compliant fashion respecting applicable local and global laws and regulations in the patient's country of residence
• The patient resides in a country in which Alexion has pursued or plans to pursue approval and reimbursement, and where Alexion or our partners have operational capabilities to safely and sustainably supply our medicine and ensure patient safety
• The patient must be under the care of a physician trained on the administration of the medicine at a suitable medical establishment