Mavorixafor

Mavorixafor
Clinical data
Trade names	Xolremdi
Other names	X4P-001; AMD-070
AHFS/Drugs.com	Multum Consumer Information
License data	US DailyMed: Mavorixafor;
Routes of; administration	By mouth
Drug class	CXCR4 antagonist
ATC code	L03AX24 (WHO) ;
Legal status
Legal status	US: ℞-only;
Identifiers
	IUPAC name N′-(1H-Benzimidazol-2-ylmethyl)-N′-[(8S)-5,6,7,8-tetrahydroquinolin-8-yl]butane-1,4-diamine;
CAS Number	558447-26-0;
PubChem CID	11256587;
IUPHAR/BPS	8580;
DrugBank	DB05501;
ChemSpider	9431613;
UNII	0G9LGB5O2W;
KEGG	D11510;
ChEBI	CHEBI:138865;
ChEMBL	ChEMBL518924;
CompTox Dashboard (EPA)	DTXSID60971247 ;
Chemical and physical data
Formula	C21H27N5
Molar mass	349.482 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C1C[C@@H](C2=C(C1)C=CC=N2)N(CCCCN)CC3=NC4=CC=CC=C4N3;
	InChI InChI=1S/C21H27N5/c22-12-3-4-14-26(15-20-24-17-9-1-2-10-18(17)25-20)19-11-5-7-16-8-6-13-23-21(16)19/h1-2,6,8-10,13,19H,3-5,7,11-12,14-15,22H2,(H,24,25)/t19-/m0/s1; Key:WVLHHLRVNDMIAR-IBGZPJMESA-N;

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]

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.^[5]

Research

Mavorixafor is in clinical trials for melanoma^[6] and renal cell carcinoma.^[7]

References

^ ^a ^b ^c ^d ^e ^f ^g "Xolremdi- mavorixafor capsule, gelatin coated". DailyMed. 4 June 2024. Retrieved 16 June 2024.
^ ^a ^b ^c ^d ^e ^f ^g ^h "FDA approves first drug for WHIM syndrome, a rare disorder". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024. This article incorporates text from this source, which is in the public domain.
^ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 30 April 2024. Retrieved 30 April 2024.
^ "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.
^ World Health Organization (2018). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 80". WHO Drug Information. 32 (3). hdl:10665/330907.
^ Andtbacka, Robert H.I.; Wang, Yan; Pierce, Robert H.; Campbell, Jean S.; Yushak, Melinda; Milhem, Mohammed; 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. PMC 10010370. PMID 36923305.
^ Choueiri, Toni K.; Atkins, Michael B.; Rose, Tracy L.; Alter, Robert S.; Ju, Yawen; Niland, Katie; 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. S2CID 231746027.

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

[Xolremdi_FDA_label-1] ^ ^a ^b ^c ^d ^e ^f ^g "Xolremdi- mavorixafor capsule, gelatin coated". DailyMed. 4 June 2024. Retrieved 16 June 2024.

[FDA_Xolremdi-2] ^ ^a ^b ^c ^d ^e ^f ^g ^h "FDA approves first drug for WHIM syndrome, a rare disorder". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024. This article incorporates text from this source, which is in the public domain.

[3] "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 30 April 2024. Retrieved 30 April 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] World Health Organization (2018). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 80". WHO Drug Information. 32 (3). hdl:10665/330907.

[6] Andtbacka, Robert H.I.; Wang, Yan; Pierce, Robert H.; Campbell, Jean S.; Yushak, Melinda; Milhem, Mohammed; 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. PMC 10010370. PMID 36923305.

[7] Choueiri, Toni K.; Atkins, Michael B.; Rose, Tracy L.; Alter, Robert S.; Ju, Yawen; Niland, Katie; 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. S2CID 231746027.

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