Expanded Access Policy

Mandos is dedicated to helping everyone affected by Niemann-Pick Type C1 (NPC1): 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.

We recognize the NPC community will leave no stone left unturned until a treatment is found and we are conducting a full evaluation of the adrabetadex* clinical data available to us. In parallel, we seek to address the urgency patients and families have expressed to us for access to adrabetadex as an investigational new drug. Adrabetadex is an investigational drug that has not been approved by FDA and FDA has not found it to be safe and effective for use to treat NPC1 or for any other use.

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. 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.

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 three to five business days.

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.