Norgine Receives European Approval for XOLREMDI (Mavorixafor), First WHIM Syndrome Treatment in EU

Key Takeaways

• XOLREMDI (mavorixafor) becomes the first authorized treatment for WHIM syndrome in the European Union
• Approval granted under exceptional circumstances for this ultra-rare primary immunodeficiency affecting fewer than 1,000 patients worldwide
• European patients with WHIM syndrome will now have access to targeted therapy following positive EMA recommendation

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Norgine announced today that the European Commission has granted marketing authorization for XOLREMDI® (mavorixafor), making it the first approved treatment for WHIM syndrome in the European Union. The approval follows a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).

The Amsterdam-based specialty pharmaceutical company received the authorization under exceptional circumstances, reflecting the ultra-rare nature of WHIM syndrome and the significant unmet medical need. WHIM syndrome is a primary immunodeficiency disorder that affects fewer than 1,000 patients worldwide, causing severe infections and increased cancer risk.

Breakthrough for Ultra-Rare Disease

WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) is caused by mutations in the CXCR4 gene, leading to neutropenia and recurrent infections. Patients typically experience chronic bacterial infections, particularly of the respiratory tract, along with persistent HPV infections causing extensive warts.

Mavorixafor works as a CXCR4 antagonist, helping to mobilize neutrophils from the bone marrow into circulation, addressing the underlying mechanism of the disease. Clinical trials demonstrated the drug’s ability to increase neutrophil counts and reduce infection rates in WHIM syndrome patients.

Market Impact and Patient Access

The European approval represents a significant milestone for the rare disease community and establishes Norgine’s position in the ultra-rare immunodeficiency market. The company previously secured FDA approval for mavorixafor in the United States, making Europe the second major market for this orphan drug.

Given the exceptional circumstances designation, the European Commission’s decision acknowledges that comprehensive clinical data may be limited due to the rarity of the condition, but the benefit-risk profile supports approval for this vulnerable patient population.

Norgine expects to begin commercial distribution across EU member states following completion of national pricing and reimbursement procedures, which typically take 6-18 months depending on the country.

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Frequently Asked Questions

What is WHIM syndrome and how many patients does it affect?

WHIM syndrome is an ultra-rare primary immunodeficiency caused by CXCR4 gene mutations, affecting fewer than 1,000 patients worldwide. It causes severe recurrent infections, warts, and low white blood cell counts.

When will XOLREMDI be available to European patients?

Following European Commission approval, XOLREMDI will become available as individual EU countries complete their pricing and reimbursement processes, typically taking 6-18 months depending on the specific country.

How does XOLREMDI work compared to current treatments?

XOLREMDI is the first targeted therapy for WHIM syndrome, working as a CXCR4 antagonist to mobilize neutrophils from bone marrow. Previously, patients only had supportive care options like antibiotics and immunoglobulin replacement therapy.