Expanded Access Policy

X4 Pharmaceuticals, Inc. Policy on Expanded Access to Investigational Drugs

X4 is committed to developing safe and effective therapies for the treatment of rare genetic disorders. Mavorixafor, is an investigational first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4 which is currently being investigated for the treatment of WHIM syndrome a rare congenital immune deficiency, characterized by Warts, Hypogammaglobulinemia, Infections, and Myelokathexis.

Expanded Access is a means by which an unapproved investigational therapy may be made available, outside of clinical trials, to treat patients with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options.  X4 understands the interest in Expanded Access programs and believes that the most appropriate way to deliver on our commitment to patients is through participation in our clinical trials.  Clinical trials, and the subsequent approval of a therapy, provide the best way to provide long-term access to as many patients as possible.  For that reason, X4 does not currently offer an expanded access program.

Patients interested in an investigational product which has not yet been approved are encouraged to speak with their doctor and potentially participate in one of X4’s current or future clinical trials.  Please find information on clinical trials by searching for X4 Pharmaceuticals at www.clinicaltrials.gov.

We recognize that not all patients will be able to enter these clinical trials. We encourage you to stay informed regarding our development programs and, as more information and clinical data on the safety and efficacy of our investigational therapies become available, our policy on expanded access will be reviewed.

If you have any questions regarding this policy, please contact: medical.affairs@x4pharma.com. X4 anticipates responding to inquiries within 7 business days.


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