Mavorixafor

Mavorixafor is an orally bioavailable CXCR4 antagonist that blocks the binding of the CXCR4 ligand, stromal-derived factor-1α (SDF-1α)/CXC Chemokine Ligand 12 (CXCL12). SDF-1/CXCR4 plays a role in trafficking and homing of leukocytes to and from the bone marrow compartment.

• XOLREMDI is indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lympocytes. XOLREMDI is a CXC chemokine receptor 4 antagonist indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.

• Use of XOLREMDI is contraindicated with drugs that are highly dependent on CYP2D6 for clearance [see Drug Interactions (7.2) ] . Use with drugs highly dependent on CYP2D6 for clearance.contraindicated

Recommended dosage: Weight more than 50 kg: 400 mg orally once daily. ( 2 ) Weight less than or equal to 50 kg: 300 mg orally once daily. ( 2 ) Administer XOLREMDI on an empty stomach after an overnight fast, and at least 30 minutes before food. ( 2 ) 2.1 Recommended Dosage The recommended dosage of XOLREMDI is: Weight more than 50 kg: 400 mg orally once daily on an empty stomach after an overnight fast, and at least 30 minutes before food. Weight less than or equal to 50 kg: 300 mg orally once daily on an empty stomach after an overnight fast, and at least 30 minutes before food. Swallow the capsules whole. Do not open, break, or chew capsules. If a dose of XOLREMDI is missed, the next dose should be taken as scheduled. Do not take more than 1 XOLREMDI dose each day. 2.2 Dosage Modifications for Strong CYP3A4 inhibitors Reduce daily dosage of XOLREMDI to 200 mg when used concomitantly with strong CYP3A4 inhibitors [see Drug Interactions (7.1) ] .

Embryo-fetal toxicity: Expected to cause fetal harm. Advise women of reproductive potential to use effective contraception. ( 5.1 , 8.1 , 8.3 ) QTc Interval Prolongation: : Correct any modifiable risk factors, assess QTc at baseline and monitor QTc during treatment as clinically indicated. XOLREMDI dose reduction or discontinuation may be required due to drug-drug interactions. ( 5.2 ) 5.1 Embryo-Fetal Toxicity Based on its mechanism of action, XOLREMDI is expected to cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.2) ] . Animal models link reductions in CXCR4/SDF-1 signaling to adverse outcomes in mammalian embryo-fetal development and to abnormal placental development. Verify the pregnancy status of female patients of reproductive potential prior to starting XOLREMDI. Advise females of reproductive potential to use an effective method of contraception during treatment with XOLREMDI and for 3 weeks after the final dose [see Use in Specific Populations (8.1 , 8.3) ] . 5.2 QTc Interval Prolongation XOLREMDI causes concentration-dependent QTc interval prolongation. QTc interval prolongation may occur when XOLREMDI is taken with concomitant medications that increase XOLREMDI exposure and/or drug products with a known potential to prolong QTc.

The following clinically significant adverse reactions are described elsewhere in the labeling: QTc Interval Prolongation [see Warnings and Precautions (5.2) ] The most common adverse reactions (›10% and at a frequency higher than placebo) were: thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact X4 Pharmaceuticals, Inc. at 1-866-MED-X4MI (1-866-633-9464) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of XOLREMDI was evaluated in Study 1, a randomized placebo-controlled trial of 31 adult and pediatric patients 12 years and older with WHIM syndrome [see Clinical Studies (14) ] . Patients received XOLREMDI 400 mg or 200 mg, based on age and body weight (N=14) or placebo (N=17). One patient received the 200 mg dose, and 13 patients received the 400 mg dose. Note that the 200 mg XOLREMDI daily dose is only recommended for use in patients receiving strong CYP3A4 inhibitors [see Dosage and Administration (2.1 , 2.2) ] .

Lactation: Advise females that breastfeeding is not recommended. ( 8.2 ) Renal Impairment: Not recommended for use in patients with severe renal impairment or end stage renal disease. ( 8.6 ) Hepatic Impairment: Not recommended for use in patients with moderate to severe hepatic impairment. ( 8.7 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action, XOLREMDI is expected to cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1) ] . There are no available data on XOLREMDI use in pregnant women informing the risk of embryo-fetal developmental toxicities. Animal models link reductions in CXCR4/SDF-1 signaling to adverse outcomes in mammalian embryo-fetal development (see Data ) . No definitive animal studies have been conducted to evaluate the effect of mavorixafor on reproduction and fetal development. Advise pregnant women of the potential risk to the fetus and to use effective contraception [see Warnings and Precautions (5.1) and Use in Specific Populations (8.3) ] . The estimated background risk of major birth defects and miscarriages for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S.

Metabolism

Mavorixafor pharmacokinetic parameters are presented as geometric mean (CV%) in adults with WHIM syndrome unless otherwise specified. Mavorixafor steady state C max is 3304 (58.6%) ng/mL and the AUC from 0 to 24 hours (AUC 0-24h ) is 13970 (58.4%) ng*h/mL following 400 mg once daily.

• Apr 26, 2024NDANDA218709X4 Pharms

1. 1Product Administration Error350%
2. 2Product Storage Error233%
3. 3Arthralgia117%
4. 4Bone Pain117%
5. 5Covid-19117%
6. 6Feeling Jittery117%
7. 7Gastrointestinal Disorder117%
8. 8Hallucination117%
9. 9Influenza117%
10. 10Nausea117%
11. 11Neutrophil Count Increased117%
12. 12Pain117%
13. 13Pneumonia Bacterial117%
14. 14Pyrexia117%
15. 15Urinary Incontinence117%

The 10 most recently updated of 15 ClinicalTrials.gov registrations naming Mavorixafor as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
  Recruiting · Phase 3 · Interventional · 176 enrolled · X4 Pharmaceuticals
  NCT06056297updated 2026-06-05
• A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
  Completed · Phase 1 · Phase 2 · Interventional · 32 enrolled · X4 Pharmaceuticals
  NCT04154488updated 2025-08-06
• Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
  Active not recruiting · Phase 3 · Interventional · 31 enrolled · X4 Pharmaceuticals
  NCT03995108updated 2025-07-29
• Drug-Drug Interaction Potential of Mavorixafor
  Completed · Phase 1 · Interventional · 38 enrolled · X4 Pharmaceuticals
  NCT06914869updated 2025-07-01
• A Study to Investigate Pharmacokinetics (PK) and Safety of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function
  Recruiting · Phase 1 · Interventional · 48 enrolled · X4 Pharmaceuticals
  NCT06858696updated 2025-04-03
• A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
  Completed · Phase 2 · Interventional · 8 enrolled · X4 Pharmaceuticals
  NCT03005327updated 2024-10-30
• Trial of X4P-001 in Participants With Advanced Renal Cell Carcinoma
  Completed · Phase 1 · Phase 2 · Interventional · 74 enrolled · X4 Pharmaceuticals
  NCT02667886updated 2024-10-03
• A Study of Mavorixafor in Combination With Ibrutinib in Participants With Waldenstrom's Macroglobulinemia (WM) Whose Tumors Express Mutations in MYD88 and CXCR4
  Completed · Phase 1 · Interventional · 16 enrolled · X4 Pharmaceuticals
  NCT04274738updated 2024-08-27
• Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma
  Terminated · Phase 1 · Phase 2 · Interventional · 9 enrolled · X4 Pharmaceuticals
  NCT02923531updated 2022-12-29
• Safety and Activity of the Oral HIV Entry Inhibitor AMD11070 in HIV Infected Patients
  Completed · Phase 1 · Phase 2 · Interventional · 6 enrolled · National Institute of Allergy and Infectious Diseases (NIAID)
  NCT00089466updated 2021-11-09

Frequently asked questions

How does Mavorixafor work?

Mavorixafor is an orally bioavailable CXCR4 antagonist that blocks the binding of the CXCR4 ligand, stromal-derived factor-1α (SDF-1α)/CXC Chemokine Ligand 12 (CXCL12). SDF-1/CXCR4 plays a role in trafficking and homing of leukocytes to and from the bone marrow compartment.

What is Mavorixafor used for?

According to FDA labeling, Mavorixafor carries indications including: XOLREMDI is indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lympocytes. XOLREMDI is a CXC chemokine receptor 4 antagonist indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Mavorixafor?

Mavorixafor is classified as Other immunostimulants, CXC Chemokine Receptor 4 Antagonist, Chemokine Receptor Type 4 Interactions, CXC Chemokine Receptor 4 Antagonists, Cytochrome P450 2D6 Inhibitors, Cytochrome P450 3A4 Inhibitors, P-Glycoprotein Inhibitors, Humoral Immunity.

What are the brand names for Mavorixafor?

Mavorixafor is marketed under brand names including Xolremdi.

What are the contraindications for Mavorixafor?

Mavorixafor labeling lists contraindications including: Use of XOLREMDI is contraindicated with drugs that are highly dependent on CYP2D6 for clearance [see Drug Interactions (7.2) ] . Use with drugs highly dependent on CYP2D6 for clearance.. Always consult the full prescribing information and a clinician.