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EQS-News: Norgine B.V.
/ Key word(s): Product Launch/Miscellaneous
Approval follows a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) EC approval marks a significant milestone for people living with this ultra-rare primary immunodeficiency and was granted under exceptional circumstances Norgine to lead commercialisation across Europe under its agreement with X4 Pharmaceuticals AMSTERDAM, April 29, 2026 /PRNewswire/ -- Norgine, a leading European specialty pharmaceutical company, today announced that the European Commission (EC) has granted marketing authorisation for mavorixafor as XOLREMDI® following a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).1 The marketing authorisation was granted under exceptional circumstances*, reflecting the ultra-rare nature of WHIM syndrome. 
XOLREMDI® is indicated in patients 12 years of age and older for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.1 WHIM syndrome is an ultra-rare primary immunodeficiency caused by dysfunction of the CXCR4 receptor, which impairs the release of white blood cells from the bone marrow into circulation. As a result, people living with the condition face an increased risk of recurrent and severe infections.2 "The approval of XOLREMDI® in the European Union marks a meaningful milestone for people living with WHIM syndrome, an ultra-rare condition that, until now, has had no approved treatment options," said Janneke van der Kamp, Chief Executive Officer, Norgine. "At Norgine, we are committed to bringing medicines that address significant unmet need to patients across Europe, Australia and New Zealand. We look forward to working with health authorities, healthcare professionals and the rare disease community to help make mavorixafor available to eligible patients as quickly as possible." The EC Marketing Authorisation is supported by results from the pivotal Phase 3 WHIM trial, a global, randomised, double-blind, placebo-controlled, 52-week multicentre study that evaluated the efficacy and safety of mavorixafor in 31 people aged 12 years and older diagnosed with WHIM syndrome.3 "This approval marks a significant moment for the WHIM syndrome community in Europe," said Johan Prevot, Executive Director forIPOPI, the International Patient Organisation for Primary Immunodeficiencies. "For patients and families living with recurrent severe infections and the burden of an ultra-rare primary immunodeficiency, the availability of an authorised treatment represents meaningful progress. We will continue to champion the patient voice across Europe and beyond to ensure that national healthcare systems recognize the value of innovative treatments and provide equitable access to patients no matter where they live." Norgine and X4 Pharmaceuticals entered into a licensing and supply agreement in January 2025, under which Norgine will commercialise mavorixafor in Europe, Australia and New Zealand following regulatory approval. All marketing authorisations in the licensed territories will be transferred to Norgine. Once completed, Norgine will be responsible for all market access and commercialisation activities in the licensed territories. X4 will manufacture and supply mavorixafor to Norgine. About WHIM Syndrome WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilisation of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections.2 About XOLREMDI® (mavorixafor) Mavorixafor is a selective CXC chemokine receptor 4 (CXCR4) antagonist that binds to the CXCR4 receptor, preventing its interaction with CXCL12,2 which is currently approved in the United States under the trade name XOLREMDI® for the treatment of WHIM syndrome.4 About Norgine Norgine is a mid-sized EU-based pharmaceutical company with 1,500 employees, generating approximately $650 million in annual sales. At Norgine, innovation drives our mission to deliver medicines that change lives. From common conditions like constipation to rare and severe diseases such as childhood cancer, we target unmet medical needs because we believe that every scientific breakthrough deserves to reach patients in need. We use our innovative development, commercialisation and manufacturing capabilities as well as strategic partnerships to navigate complex pathways. Combined with our extensive history and deep regional expertise, this approach allows us to accelerate and expand the reach of life-changing medicines across Europe, Australia, and New Zealand. We are guided by the trust that healthcare professionals and patients place in us and remain committed to delivering innovation that transforms lives, one patient at a time. *** *European Union definition of "under exceptional circumstances": A type of marketing authorisation granted where the applicant is unable to provide comprehensive data on the efficacy and safety, as is often the case in ultra-rare conditions. In such cases the benefit–risk is assessed based on the available data, with additional data required following approval in accordance with regulatory requirements. References: 1. EMA. XOLREMDI. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/xolremdi. Logo - https://mma.prnewswire.com/media/2846726/5944580/Norgine_Logo.jpg 
29.04.2026 CET/CEST Dissemination of a Corporate News, transmitted by EQS News - a service of EQS Group. |
View original content:2318020 29.04.2026 CET/CEST