Mavorixafor for WHIM Syndrome Receives CHMP Support
Norgine has welcomed a positive CHMP opinion recommending approval of mavorixafor for WHIM syndrome in the European Union. The opinion was issued by the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP).
The recommendation supports granting marketing authorisation under exceptional circumstances for XOLREMDI® (mavorixafor). The treatment is intended for patients aged 12 years and older diagnosed with WHIM syndrome. Importantly, it aims to increase circulating mature neutrophils and lymphocytes.
Addressing an Unmet Need in WHIM Syndrome
WHIM syndrome is a rare primary immunodeficiency disorder. It stands for warts, hypogammaglobulinemia, infections and myelokathexis. The disease results from dysfunction of the CXCR4 chemokine receptor. As a result, white blood cells remain trapped in the bone marrow. Consequently, patients experience recurrent or severe infections.
Currently, there is no other licensed treatment option for this condition in the EU. Therefore, the positive CHMP opinion marks a significant regulatory milestone. It brings hope to patients and families affected by WHIM syndrome.
European Commission Decision Expected in Q2 2026
Following the positive CHMP opinion, the European Commission will review the recommendation. A final decision on mavorixafor for WHIM syndrome is expected in the second quarter of 2026.
If approved, Norgine will lead commercialisation efforts across Europe, Australia, and New Zealand. This follows its licensing and supply agreement with X4 Pharmaceuticals signed in January 2025. Under the agreement, Norgine will handle market access and commercial activities in the licensed territories. Meanwhile, X4 will manufacture and supply the medicine.
Phase 3 Data Supports Approval
The CHMP recommendation is based on results from the global Phase 3 4WHIM trial. This randomized, double-blind, placebo-controlled study evaluated 31 patients aged 12 years and older. The trial assessed both efficacy and safety over 52 weeks.
Overall, the data demonstrated clinically meaningful improvements in immune cell counts. Therefore, the study provided strong support for regulatory review.
This positive CHMP opinion for mavorixafor represents a major step toward improving care for people living with WHIM syndrome. Moreover, it highlights Norgine’s commitment to delivering innovative rare disease treatments across its regions.
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