AI Article Synopsis
- - Mavorixafor (XOLREMDI™) is an oral medication that blocks the CXCR4 receptor to treat WHIM syndrome, a rare genetic disorder characterized by warts, low immunity, and infections.
- - It received its first approval in the USA in April 2024 for use in patients 12 years and older, helping to boost the levels of neutrophils and lymphocytes in the blood.
- - Ongoing research is exploring its potential benefits for other chronic neutropenic disorders, with the article detailing its developmental milestones leading to its FDA approval.
Article Abstract
Mavorixafor (XOLREMDI™) is an oral, selective C-X-C chemokine receptor 4 (CXCR4) antagonist developed by X4 Pharmaceuticals that blocks the binding of C-X-C chemokine ligand 12 (also known as stromal derived factor-1) to CXCR4. In April 2024, it became the first therapy to be approved for WHIM syndrome (named by an acronym for its observed characteristics of Warts, Hypogammaglobulinaemia, Infections and Myelokathexis) in the USA, where it is indicated for use in patients aged ≥ 12 years with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. Clinical development of mavorixafor is ongoing for chronic neutropenic disorders. This article summarizes the milestones in the development of mavorixafor leading to this first approval for use in patients aged ≥ 12 years with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1007/s40265-024-02063-y | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Elevated CXCL1 triggers dopaminergic neuronal loss in the substantia nigra of C57BL/6J mice: Evaluation of a novel Parkinsonian mouse model.
Zool Res
January 2025
Institute of Brain Science and Disease, School of Basic Medicine, Shandong Provincial Key Laboratory of Pathogenesis and Prevention of Neurological Disorders, Qingdao University, Qingdao, Shandong 266071, China. E-mail:
Substantial evidence points to the early onset of peripheral inflammation in the development of Parkinson's disease (PD), supporting the "body-first" hypothesis. However, there remains a notable absence of PD-specific animal models induced by inflammatory cytokines. This study introduces a novel mouse model of PD driven by the proinflammatory cytokine CXCL1, identified in our previous research.
View Article and Find Full Text PDFEffects of initial periodontal therapy on the formation of neutrophil extracellular traps in gingival crevicular fluid in patients with severe periodontitis.
Hua Xi Kou Qiang Yi Xue Za Zhi
February 2025
Dept. of Periodontology, The Affiliated Stomatological Hospital of Nanjing Medical University & State Key Laboratory Cultivation Base of Research, Prevention and Treatment for Oral Diseases & Jiangsu Province Engineering Research Center of Stomatological Translational Medicine, Nanjing 210029, China.
Objectives: This study aimed to observe the effects of initial periodontal therapy on the level of neutrophil extracellular traps (NETs) in gingival crevicular fluid (GCF) of patients with severe periodontitis and to analyze the factors related to the formation of NETs.
Methods: Thirty-one patients with stage Ⅲ-Ⅳ periodontitis were recruited. Clinical periodontal parameters, including plaque index (PLI), gingival index (GI), probing depth (PD), and clinical atta-chment loss (CAL), were recorded before and 6-8 weeks after initial periodontal therapy.
C-X-C chemokine receptor type 5CD8 T cells as immune regulators in hepatitis Be antigen-positive chronic hepatitis B under interferon-alpha treatment.
World J Gastroenterol
January 2025
Institute of Hepatology and Department of Infectious Diseases, The Second Xiangya Hospital, Central South University, Changsha 410011, Hunan Province, China.
Background: C-X-C chemokine receptor type 5 (CXCR5)CD8 T cells represent a unique immune subset with dual roles, functioning as cytotoxic cells in persistent viral infections while promoting B cell responses. Despite their importance, the specific role of CXCR5CD8 T cells in chronic hepatitis B (CHB), particularly during interferon-alpha (IFN-α) treatment, is not fully understood. This study aims to elucidate the relationship between CXCR5CD8 T cells and sustained serologic response (SR) in patients undergoing 48 weeks of pegylated IFN-α (peg-IFN-α) treatment for CHB.
View Article and Find Full Text PDFThe role of IFN-γ/CXCL10 axis in Mycoplasma pneumonia infection.
Sci Rep
January 2025
Department of Respiratory Medicine, Children's Hospital of Soochow University, Suzhou, China.
Mycoplasma pneumoniae caused lower respiratory tract infection in children and can exacerbate these infections through the production of various inflammatory factors, with chemokines playing a key role. However, the pathogenesis of this infection is complicated and thus has not been thoroughly studied. We clarified that cytokine expression levels were analyzed in both peripheral blood and bronchoalveolar lavage fluid (BALF), and in vitro assays were conducted using THP-1 macrophages.
View Article and Find Full Text PDFTRAF2 and RIPK1 redundantly mediate classical NFκB signaling by TNFR1 and CD95-type death receptors.
Cell Death Dis
January 2025
Division of Molecular Internal Medicine, Department of Internal Medicine II, University Hospital Würzburg, Auvera Haus Grombühlstraße 12, 97080, Würzburg, Germany.
This study suggests a modified model of TNFR1-induced complex I-mediated NFκB signaling. Evaluation of a panel of five tumor cell lines (HCT116-PIK3CAmut, SK-MEL-23, HeLa-RIPK3, HT29, D10) with TRAF2 knockout revealed in two cell lines (HT29, HeLa-RIPK3) a sensitizing effect for death receptor-induced necroptosis and in one cell line (D10) a mild sensitization for TNFR1-induced apoptosis. TRAF2 deficiency inhibited death receptor-induced classical NFκB-mediated production of IL-8 only in a subset of cell lines and only partly.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!