European Commission approves Norgine’s XOLREMDI® (mavorixafor) for WHIM syndrome in patients aged 12+, supported by Phase 3 data, marking the first authorised therapy for the ultra-rare condition.
Written By: Mahathi Palivela, PharmD
Reviewed By: Pharmacally Editorial Team
The European Commission has granted marketing authorisation for mavorixafor, marketed as XOLREMDI®, for the treatment of WHIM syndrome in patients aged 12 years and older. The approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency and has been issued under exceptional circumstances due to the ultra-rare nature of the disease.
Mavorixafor is indicated to increase circulating mature neutrophils and lymphocytes in patients with WHIM syndrome, a rare primary immunodeficiency characterised by warts, hypogammaglobulinemia, infections, and myelokathexis.
The condition is caused by dysfunction of the CXCR4 receptor, which impairs the release of white blood cells from the bone marrow into circulation. As a result, affected individuals experience neutropenia and lymphopenia, leading to recurrent and severe infections.
The approval is supported by results from a pivotal Phase 3 trial (NCT03995108), a global, randomised, double-blind, placebo-controlled, 52-week multicentre study involving 31 patients aged 12 years and older with WHIM syndrome. The study evaluated the efficacy and safety of mavorixafor.
Company and patient representatives highlighted the significance of the approval. Janneke van der Kamp, Chief Executive Officer of Norgine, described the decision as an important step for patients with WHIM syndrome, noting the absence of previously approved treatment options and the company’s intent to work with stakeholders to enable timely access.
Johan Prevot, Executive Director of the International Patient Organisation for Primary Immunodeficiencies, stated that the availability of an authorised therapy represents meaningful progress for patients and families managing the burden of recurrent infections and emphasised the need for equitable access across healthcare systems.
Mavorixafor is a selective CXCR4 antagonist that blocks interaction with its ligand CXCL12, promoting the mobilization of white blood cells into circulation. The therapy is already approved in the United States under the same brand name for the same indication of WHIM syndrome.
Under a licensing and supply agreement signed in January 2025, Norgine will commercialize mavorixafor in Europe, Australia, and New Zealand, while X4 Pharmaceuticals will manufacture and supply the product. Following the transfer of marketing authorizations, Norgine will be responsible for market access and commercialization activities in these regions.
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About the Writer
Mahathi Palivela is pursuing PharmD and has a strong interest in Clinical Pharmacy and Patient safety. She is passionate about handling and analyzing patient data, and translating clinical insights into clear, meaningful summaries. She aims to apply this interest in Medical Writing and Pharmacovigilance, focusing on improving patient outcomes through careful data interpretation and communication.