Die US-amerikanische FDA genehmigt die Therapie von X4 Pharmaceuticals für das WHIM-Syndrom
https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-x4-pharmaceuticals-drug-immunodeficiency-disease-2024-04-29/
Die US-amerikanische FDA genehmigt die Therapie von X4 Pharmaceuticals für das WHIM-Syndrom
https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-x4-pharmaceuticals-drug-immunodeficiency-disease-2024-04-29/
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> X4’s mavorixafor, to be sold under the brand name Xolremdi, is the first therapy to get U.S. approval specifically for the treatment of WHIM syndrome.
> WHIM is characterized by disorders in which the body’s immune system does not function properly. It is an acronym for warts, hypogammaglobulinemia or low antibody levels, infections, and myelokathexis or low white blood cell count, which are all symptoms of the disorder.
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> The approval is based on a late-stage study where the therapy showed 60% reduction in infection rate compared to placebo when tested in patients with WHIM syndrome.
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> Less than 1,000 people in the United States have WHIM syndrome, according to government data.