Mahzi Therapeutics, Inc. (Mahzi) is committed to advancing innovative therapies for patients with under-served rare neurogenetic disorders. We recognize that some patients with unmet medical needs may not be eligible to participate in our clinical trials and may have no other treatment alternatives available. Mahzi may consider requests for access to our investigational medicine(s) outside of a clinical trial setting through a process commonly referred to as compassionate use or expanded access.
Mahzi’s investigational therapies have not received marketing approval from the US Food and Drug Administration (FDA) or any other regulatory authority. The safety and efficacy profiles of investigational therapies have not been fully established. Physicians and their patients should carefully weigh all potential risks and benefits before pursuing expanded access.
Mahzi will evaluate each request based upon many factors including local regulatory requirements; stage of clinical development; current supply constraints; internal policies; and medical and scientific assessments.
Mahzi is not obligated to provide investigational medicines through expanded access and reserves the right to decline requests. Any provision of investigational medicine under this program will be subject to, at a minimum, required health authority and ethics committee approvals, execution of appropriate agreements and informed consent documentation, and adherence to physician data collection and safety reporting obligations. Mahzi will assume no responsibility for administration of any medicines, or ancillary care costs for a treatment under a compassionate use/expanded access approval.
For Healthcare Professionals:
If a clinical trial is not a viable option for your patient and no satisfactory alternative treatments exist, you may submit a request for expanded access on behalf of your patient by contacting us at: EAP@mahzi.com.
For Caregivers and/or Patients:
If you believe you or your child may be a candidate for compassionate use/expanded access, speak with your treating physician about initiating a request on your behalf. Mahzi only reviews requests submitted directly by a licensed physician.