Mavorixafor

Mavorixafor, sold under the brand name Xolremdi, is a medication used for the treatment of WHIM syndrome.[1] It is a CXC chemokine receptor 4 antagonist.[1] It is taken by mouth.[1] It was developed by X4 Pharmaceuticals.

The most frequently reported adverse reactions include thrombocytopenia (low platelet counts), rash, rhinitis (stuffy nose), epistaxis (nosebleed), vomiting, and dizziness.[2]

Mavorixafor was approved for medical use in the United States in April 2024.[1][2][3][4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]

## Contents

* 1 Medical uses
* 2 Adverse effects
* 3 History
* 4 Society and culture
* 4.1 Names
* 5 Research
* 6 References
* 7 External links

## Medical uses

Mavorixafor is indicated in people twelve years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.[1][2]

## Adverse effects

Mavorixafor is expected to cause harm to the baby.[1][2]

## History

The effectiveness of mavorixafor was evaluated in a 52-week, randomized, double-blind, placebo-controlled trial that enrolled 31 adolescents and adults with WHIM syndrome (NCT03995108).[2] Mavorixafor improved absolute neutrophil counts and absolute lymphocyte counts, assessed over a 24-hour period four times throughout the study.[2] Absolute neutrophil counts below 500 cells/μL and absolute lymphocyte counts below 1000 cells/μL are associated with an increased risk of infections.[2] The average length of time over 24 hours that counts were above these levels was significantly longer with mavorixafor compared to the placebo group (15.0 hours compared to 2.8 hours for absolute neutrophil counts; 15.8 hours compared to 4.6 hours for absolute lymphocyte counts).[2]

## Society and culture

### Names

Mavorixafor is the international nonproprietary name.[6]

## Research

Mavorixafor is in clinical trials for melanoma[7] and renal cell carcinoma.[8]

## References

1. ↑ 1.0 1.1 1.2 1.3 1.4 1.5 Cite error: Invalid `<ref>` tag; no text was provided for refs named `Xolremdi FDA label`
2. ↑ 2.0 2.1 2.2 2.3 2.4 2.5 2.6 2.7 "FDA approves first drug for WHIM syndrome, a rare disorder". 29 April 2024. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-drug-whim-syndrome-rare-disorder-can-lead-recurrent-life-threatening-infections. This article incorporates text from this source, which is in the public domain.
3. ↑ "Novel Drug Approvals for 2024". 29 April 2024. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024.
4. ↑ "X4 Pharmaceuticals Announces FDA Approval of Xolremdi (mavorixafor) Capsules, First Drug Indicated in Patients with WHIM Syndrome" (Press release). X4 Pharmaceuticals. 29 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024 - via GlobeNewswire.
5. ↑ (PDF) ﻿New Drug Therapy Approvals 2024﻿ (Report). January 2025. https://www.fda.gov/media/184967/download. Retrieved 21 January 2025.
6. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 80". WHO Drug Information 32 (3). 2018.
7. ↑ Andtbacka, Robert H.I.; Wang, Yan; Pierce, Robert H.; Campbell, Jean S.; Yushak, Melinda; Milhem, Mohammed; Ross, Merrick; Niland, Katie et al. (31 August 2022). "Mavorixafor, an Orally Bioavailable CXCR4 Antagonist, Increases Immune Cell Infiltration and Inflammatory Status of Tumor Microenvironment in Patients with Melanoma". Cancer Research Communications 2 (8): 904–913. doi:10.1158/2767-9764.CRC-22-0090. PMID 36923305.
8. ↑ Choueiri, Toni K.; Atkins, Michael B.; Rose, Tracy L.; Alter, Robert S.; Ju, Yawen; Niland, Katie; Wang, Yan; Arbeit, Robert et al. (August 2021). "A phase 1b trial of the CXCR4 inhibitor mavorixafor and nivolumab in advanced renal cell carcinoma patients with no prior response to nivolumab monotherapy". Investigational New Drugs 39 (4): 1019–1027. doi:10.1007/s10637-020-01058-2. PMID 33507454.

## External links

* Clinical trial number NCT03995108 for "Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome" at ClinicalTrials.gov

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Immunostimulants (L03)

Endogenous|

| Cytokines|

| Colony-stimulating factors|

* G-CSF
* Filgrastim / Pegfilgrastim
* Lenograstim
* GM-CSF
* Molgramostim
* Sargramostim
* SCF
* Ancestim

|

Interferons|

* alpha:
* Albinterferon
* Interferon alfa natural
* Interferon alfa 2a / Peginterferon alfa-2a
* Interferon alfa 2b / Peginterferon alfa-2b
* Interferon alfa n1
* Interferon alfacon-1
* Interferon alpha-n3
* beta:
* Interferon beta natural
* Interferon beta 1a
* Interferon beta 1b
* Interferon gamma

Interleukins|

* Aldesleukin
* Oprelvekin

Other protein / peptide|

* Growth hormone
* Immunocyanin
* Pegademase
* Prolactin
* Tasonermin

Other|

* Female sex steroids
* Histamine dihydrochloride
* Poly ICLC
* Vitamin D

Exogenous|

* beta-glucan
* Lentinan
* heterocyclic compound
* Plerixafor
* hydroxyquinoline
* Roquinimex
* Mifamurtide
* oligopeptides
* Glatiramer acetate
* Thymopentin
* Thymosin α1
* Thymulin
* polyribonucleotide
* Polyinosinic:polycytidylic acid
* thiazolidine
* Pidotimod
* vaccines
* Bacillus Calmette–Guérin
* Melanoma vaccine
* Sipuleucel-T

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