Expanded Access Policy
Mandos is dedicated to helping everyone affected by Neimann-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.
Transparency: How we consider requests for Expanded Access
While we strive to provide access to patients with unmet medical need, decisions about requests for expanded access to an investigational drug can be complex and challenging. We work to make these decisions in a fair and equitable manner, taking into consideration the specifics of the patient’s case, the available medical and scientific information about the investigational medicine, and the probability and timing of a potential regulatory approval.
For Mandos to consider an expanded access program request, the following criteria should first be met:
- The patient’s licensed physician submits an unsolicited request for expanded access
- The patient has a serious or life-threatening illness and fulfills objective clinical criteria establishing that the patient is in critical need
- The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated
- There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition
- The patient is ineligible for, or otherwise unable to, participate in a clinical trial for the investigational drug
- There is adequate supply of the investigational drug
- The investigational drug can be properly administered – and it is logistically feasible to make it available – outside of the clinical trial setting
- Expanded access is permitted under applicable law
Once all the necessary information listed above has been received by Mandos, the request will be carefully evaluated by the Mandos team. We will will make every effort to provide a timely decision and may request more detailed information from the physician or institution in order to fully evaluate a request. Decisions will be made based on scientific evidence available to the company at the time of the request and in consideration of applicable local laws and regulations.
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 make a formal request on behalf of the 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.