Expended Access

A diagnosis of a rare or ultra-rare disease can profoundly alter the lives of patients and their families. We recognize the responsibility to thoughtfully support patients as they navigate complex and often limited treatment options. As we work to develop new therapies for children and adults with rare conditions, our goal is to ensure that investigational medicines, those not yet approved by regulatory authorities such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA), are made available through appropriate and established pathways.

 

Participation in a clinical trial remains the primary and preferred means of accessing our investigational therapies. However, we recognize that some patients with serious or immediately life-threatening conditions may not qualify for, or have access to, a clinical study and may have no satisfactory therapeutic alternatives. In select cases of critical and compelling medical need, we may consider providing access to an investigational therapy through our Expanded Access Program.

 

Availability under this program is determined on a case-by-case basis and is contingent upon multiple factors, including drug supply, available funding, the clinical team capabilities and readiness of the treating hospital, and the severity and urgency of the patient’s condition.