Norgine Receives European Commission Approval for XOLREMDI® Treatment for WHIM Syndrome

Norgine Secures Approval for XOLREMDI®

Norgine, a leading European specialty pharmaceutical company, has announced a significant milestone in its mission to provide innovative healthcare solutions. Recently, the European Commission granted marketing authorization for XOLREMDI®, an impactful treatment for patients suffering from the ultra-rare WHIM syndrome. This approval follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

The WHIM syndrome, characterized by a range of symptoms including warts, recurrent infections, and hypogammaglobulinemia due to neutropenia, is caused by a dysfunction in the CXCR4 receptor, which hinders the release of white blood cells from the bone marrow. As a result, patients face a heightened risk of severe and recurrent infections.

Norgine's CEO, Janneke van der Kamp, emphasized the importance of this approval, noting that until now, patients with WHIM syndrome had no approved treatment options. She stated, "This mark signifies a crucial step forward for those affected by WHIM syndrome, offering them an effective treatment path that was previously unavailable. Our mission at Norgine is to deliver innovative medications to meet significant unmet needs, and we are eager to collaborate with healthcare authorities and professionals to ensure prompt access to XOLREMDI® for eligible patients."

XOLREMDI® targets patients aged 12 years and older, aiming to increase the levels of circulating mature neutrophils and lymphocytes, thereby improving immune function. The market authorization is further substantiated by data from a pivotal Phase 3 clinical trial, which demonstrated the safety and efficacy of mavorixafor (the generic name for XOLREMDI®) in patients diagnosed with WHIM syndrome.

This milestone is not only essential for patients but also holds significance for the broader community, as noted by Johan Prevot, Executive Director of IPOPI, the International Patient Organization for Primary Immunodeficiencies. Prevot remarked, "The availability of an approved treatment represents substantial progress for patients and families grappling with the burden of this ultrarare immune deficiency. We will continue advocating for patient voices across Europe, ensuring that health systems recognize the value of innovative treatments, providing equitable access for all patients."

In January 2025, Norgine and X4 Pharmaceuticals entered into a licensing agreement, allowing Norgine to market mavorixafor in Europe, Australia, and New Zealand post-regulatory approval. With this agreement, Norgine assumes responsibility for all market access and commercialization activities within the licensed territories, while X4 will manufacture and supply the drug.

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency characterized by chronic neutropenia, primarily due to a malfunction in the CXCR4 receptor. Its name derives from the four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis. Most patients present with low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), leading to severe or frequent infections.

About XOLREMDI® (Mavorixafor)

Mavorixafor is a selective CXCR4 receptor antagonist that works by preventing the receptor's interaction with its ligand CXCL12, potentially addressing the underlying cause of WHIM syndrome. It is already approved in the U.S. for the same purpose under the name XOLREMDI®.

About Norgine

Norgine operates as a mid-sized pharmaceutical firm across Europe, employing around 1,500 people and generating an annual revenue of approximately USD 650 million. Norgine emphasizes innovation in its operations, aiming to deliver life-changing medications that cater to both common and rare medical conditions. The company's commitment lies in addressing unmet medical needs through strategic development and collaboration efforts, enabling patients in Europe, Australia, and New Zealand to access vital treatments soonest.

This approval not only highlights Norgine's dedication but also represents hope for patients facing the challenges posed by WHIM syndrome, allowing for improved health outcomes and quality of life.