Managed Access Programs
Consistent with the Marinus mission to bring innovative medicines to patients with serious or life-threatening conditions, we are focused on conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. Clinical trials are an essential part of collecting the necessary data on safety and efficacy to support regulatory approval. At the same time, we understand that there are patients who will not be eligible for our clinical trials and for whom there are limited options for alternative therapies.
To address the needs of individual patients with a serious or life-threatening condition who will not be eligible for our clinical trials and for whom there are limited options for alternative therapies, Marinus has two distinct managed access programs available to treating physicians for individual eligible patients, the Marinus Access Program and the Compassionate Use Program.
About the Marinus Access Program
The Marinus Access Program enables physicians to request ZTALMY® (ganaxolone) oral suspension CV for eligible patients with seizures associated with CDKL5 deficiency disorder in geographies where the product is not commercially available and as supported by local regulatory requirements. The program does not provide free of charge access to ZTALMY and does not affect patients already taking ZTALMY as part of a clinical trial.
The Marinus Access Program is managed by Uniphar, a leader in the international distribution of specialized pharmaceuticals. Physicians can learn more about the Marinus Access Program for ZTALMY by sending an email to MarinusMAP@Uniphar.com.
Please note: Medication access requests in the following geographies where Marinus has a commercial arrangement in place are managed by the local commercial organization and are not eligible for the Marinus Access Program:
- European Economic Area, United Kingdom, Switzerland: Orion Corporation
- Algeria, Bahrain, Egypt, Iraq, Jordan, Kingdom of Saudi Arabia, Kuwait, Lebanon, Libya, Morocco, Oman, Qatar, Tunisia, and United Arab Emirates: Biologix FZco
- Mainland China, the Hong Kong Special Administrative Region, Macau Special Administrative Region, and Taiwan: Tenacia Biotechnology Co., Ltd.
About the Compassionate Use Program
Marinus will consider providing a physician with pre-approval access to a Marinus investigational product under a Compassionate Use Program for the treatment of an individual patient outside of a clinical trial under certain conditions.
These conditions include the following:
– The patient has a serious or life-threatening illness or condition and has no comparable or satisfactory alternative therapy available;
– The patient is ineligible for enrollment into or unable to access ongoing clinical trials;
– A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
– The patient meets any other important medical criteria established by the medical experts working on the product development program;
– Making the investigational product available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational product;
– Adequate supply of the investigational product is available; and
– Such access is allowed as per local laws and regulations.
We continually evaluate the benefit-risk profile of our investigational products based on evolving clinical data and may make changes to this Compassionate Use Policy when appropriate.
Marinus is committed to evaluating all requests in a fair and equitable manner by qualified Marinus experts. All requests must be submitted by the patient’s treating physician. Marinus may require more detailed information to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by the Marinus review team whose decisions are final.
Physicians seeking pre-approval access through a Compassionate Use Program for individual patients with no alternative treatment options should submit their requests to MedInfo@marinuspharma.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within three (3) business days after receipt.