Expanded Access Policy
Mandos is dedicated to helping everyone affected by Niemann-Pick disease type C (NPC): patients, parents, carers, physicians, and communities. We were purpose-built for patients and recognize the community’s urgency for both progress and access. We collaborate with stakeholders to more deeply understand what’s most important and how we can build a path forward.
Expanded Access Programs are intended for patients with serious or life-threatening conditions who request access to investigational products that are not approved in their country and are unable to participate in ongoing clinical trials.
We understand that there may be cases when it is not possible for a patient to participate in a clinical trial, the patient is in critical medical need, and all other available medical options have been exhausted. In those cases, the patient’s physician may seek special access to a Mandos investigational drug outside of the clinical trial setting. These access programs may be called expanded access, early access, compassionate use, emergency use, or other similar terms.
If you’re considering enrolling in an Expanded Access Program, please contact your primary care provider and they will make a determination of eligibility based on FDA standards.
How to Request Expanded Access
We encourage patients to speak first with their treating physician about their eligibility to enroll in a clinical trial. If a treating physician believes expanded access may be the only option for a patient, the physician should contact Mandos to discuss the Expanded Access Program process for your patient.
To ensure the safety of patients, Mandos has developed criteria to evaluate a physician's request on an individual patient basis. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all relevant laws, regulations, and requirements, including safety, monitoring, patient consent, and other requirements defined by Mandos. Requests for expanded access may only be made by licensed physicians with expertise in the disease, who understand the potential risks and benefits of this investigational drug and agrees to comply with Mandos’ Expanded Access policy.
Please contact Mandos if you have questions about or requests for expanded access to adrabetadex. You may expect to receive an acknowledgment of receipt of your inquiry within approximately two business days.
Contact Submission Form
As authorized by the 21st Century Cures Act, Mandos may revise this Expanded Access policy at any time. Additionally, the availability of this policy shall not serve as a guarantee of access to any specific investigational drug by any individual patient.