Expanded Access Program (EAP)
Clinical trials and the subsequent approval of a therapy provide the best way to make therapies accessible to as many patients as possible. X4 Pharmaceuticals’ sole registered medicine, mavorixafor (XOLREMDI®) is currently only approved in the United States for patients with WHIM Syndrome. Mavorixafor is not approved in any country for primary chronic neutropenia.
Patients interested in any investigational product that has not yet been approved are encouraged to speak with their doctor and potentially participate in our ongoing clinical trials, which can be found by searching for X4 Pharmaceuticals at www.clinicaltrials.gov (in the United States) and https://euclinicaltrials.eu/search-clinical-trials-reports (in the European Union).
Currently, we will consider granting expanded access to our investigational medicinal products only if the following criteria are met:
- The patient has been diagnosed with WHIM syndrome, resides outside of the United States, and was enrolled in an X4-sponsored long-term extension of a trial at the time that trial was completed;
- There are no comparable or satisfactory alternative therapies available for the patient;
- The patient is ineligible for, or otherwise unable to, participating in a clinical trial;
- There is sufficient evidence to believe that the potential benefit from use of the investigational medicine outweighs the potential risk to the patient;
- X4 has adequate supply of the investigational medicine and providing the investigational medicine for expanded access will not interfere with ongoing clinical trial(s) or with the overall development program; and
- Such access provision is allowed per applicable national laws, regulations, and requirements.
A request for expanded access must be initiated by the treating physician through a written application. Applications are available from our Medical Information team at medicalinfo@x4pharma.com. Applications will be acknowledged within approximately five (5) business days upon receipt. Please note that a submission does not guarantee a patient will have access. No access to investigational medical products, other than participation in ongoing clinical trials, is currently available for patients with primary chronic neutropenia in any country.
We encourage you to stay informed regarding our development programs and we will continue to review our expanded access policy as needed.
For additional information, physicians with eligible patients can contact our Medical Information team at medicalinfo@x4pharma.com.