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Norgine Welcomes CHMP's Positive Opinion on Mavorixafor for WHIM Syndrome Treatment

Norgine B.V., a prominent European specialty pharmaceutical company, has officially welcomed the favorable recommendation from the Committee for Medicinal Products for Human Use (CHMP) regarding the conditional marketing authorization of Mavorixafor for the treatment of patients aged 12 years and older diagnosed with WHIM syndrome (Warts, Hypogammaglobulinaemia, Infections, and Myelokathexis). This step is crucial as it could lead to the first official treatment available for this condition in the European Union.

Significance of the CHMP Recommendation

The CHMP’s recommendation signifies a monumental regulatory milestone for the numerous patients suffering from WHIM syndrome, who currently lack any approved therapeutic options. This rare genetic disorder is characterized by a malfunction in the CXCR4 chemokine receptor which inhibits the proper release of white blood cells from the bone marrow into the bloodstream, resulting in a heightened susceptibility to recurrent and severe infections.

The European Commission (EC) is now tasked with reviewing the CHMP's recommendation, with a definitive decision anticipated in the second quarter of 2026. Janneke van der Kamp, CEO of Norgine, expressed optimism regarding this decision, emphasizing the importance of moving forward for patients affected by WHIM syndrome. “We are excited about this critical regulatory advance for those living with WHIM syndrome,” she stated. “At Norgine, we have built a comprehensive expertise in providing rare and specialty medicines across Europe, Australia, and New Zealand, and the positive CHMP opinion for Mavorixafor reflects the kind of innovation our company aims to deliver for patients in need.”

Clinical Evidence Supporting Mavorixafor

The recommendation from the CHMP is informed by the results from the pivotal Phase 3 clinical trial (4WHIM). This global, randomized, double-blind, placebo-controlled study, conducted over 52 weeks, involved 31 participants aged 12 and older, assessing the efficacy and safety of Mavorixafor specifically for WHIM syndrome. The findings from this study highlight the potential benefits of Mavorixafor in increasing the number of circulating mature neutrophils and lymphocytes in patients, which is critical for their immune functioning.

Norgine and X4 Pharmaceuticals entered into a licensing and supply agreement in January 2025, which will allow Norgine to market Mavorixafor in Europe, Australia, and New Zealand following authorization. Under this arrangement, Norgine will manage all market access and commercialization activities in the areas covered, while X4 Pharmaceuticals will take on the manufacturing and delivery responsibilities for Mavorixafor.

Understanding WHIM Syndrome

WHIM syndrome is an exceptionally rare condition defined by a combined primary immunodeficiency and chronic neutropenia, resulting from a dysfunction of the CXCR4 receptor. This leads to impaired mobilization of white blood cells from the bone marrow into the peripheral circulation. The condition is named after its four hallmark features: warts, hypogammaglobulinaemia, infections, and myelokathexis; however, it is critical to note that not every patient displays all four symptoms. Individuals affected by WHIM syndrome typically present with low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), making them highly vulnerable to severe and/or frequent infections.

A Closer Look at Mavorixafor

Mavorixafor functions as a selective CXCR4 antagonist that binds to the CXCR4 receptor, obstructing its interaction with CXCL12, thus enhancing white blood cell release into circulation. In the United States, Mavorixafor is already approved for WHIM syndrome under the name XOLREMDI.

About Norgine

Norgine is a mid-sized pharmaceutical company based in the EU, employing around 1,500 people and generating an annual revenue of approximately $650 million. The company is driven by a mission to deliver innovative therapies that make a difference in patients’ lives. They focus on a wide range of medical needs, from common ailments like constipation to rare and serious conditions such as childhood cancers. Norgine aims to expedite the reach of life-changing medicines through strong development, marketing, and manufacturing capabilities, coupled with strategic partnerships.

Through this unwavering dedication, they continue to earn and uphold the trust of healthcare professionals and patients alike, striving to create innovations that positively impact the lives of each and every patient.