Norgine B.V. has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommending the granting of a marketing authorisation under exceptional circumstances for XOLREMDI (mavorixafor). The therapy is indicated for patients aged 12 years and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.

WHIM syndrome — characterised by warts, hypogammaglobulinemia, infections and myelokathexis — is a rare primary immunodeficiency caused by dysfunction of the CXCR4 receptor. The condition disrupts the release of white blood cells from the bone marrow into circulation, resulting in recurrent and often severe infections. Currently, there are no licensed treatment options specifically approved for this disorder.

The CHMP’s recommendation represents a significant regulatory milestone for patients living with WHIM syndrome. The opinion will now be reviewed by the European Commission, with a final decision on marketing authorisation expected in the second quarter of 2026.

The positive opinion is supported by data from the pivotal Phase III 4WHIM trial, a global, randomised, double-blind, placebo-controlled, 52-week multicentre study involving 31 patients aged 12 years and older diagnosed with WHIM syndrome. The study evaluated the efficacy and safety of mavorixafor and demonstrated clinically meaningful improvements in white blood cell counts.

Under a licensing and supply agreement signed in January 2025, X4 Pharmaceuticals granted Norgine exclusive rights to commercialise mavorixafor across Europe, Australia and New Zealand following regulatory approval. Marketing authorisations in the licensed territories will be transferred to Norgine, which will oversee market access and commercialisation, while X4 Pharmaceuticals will manufacture and supply the product.

If approved by the European Commission, XOLREMDI would become the first authorised treatment specifically indicated for WHIM syndrome in the region.