WHIM syndrome

Active Ingredient: Mavorixafor

Indication for Mavorixafor

Population group: only adolescents (12 years - 18 years old) , adults (18 - 65 years old)

Therapeutic intent: Curative procedure

Mavorixafor 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 lymphocytes.

For this indication, competent medicine agencies globally authorize below treatments:

For patients weighting >50 kg 400 mg once daily and for patients weighting ≤50 kg 300 mg once daily

For:

Dosage regimens

Regimen A: In case that patient weight is ≥ 50 kg, oral, 400 milligrams mavorixafor, once daily -30 minutes before breakfast.

Regimen B: In case that patient weight is ≤ 50 kg, oral, 300 milligrams mavorixafor, once daily -30 minutes before breakfast.

Detailed description

The recommended dosage of mavorixafor 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.

If a dose of mavorixafor is missed, the next dose should be taken as scheduled. Do not take more than one mavorixafor dose each day.

Dosage considerations

Avoid eating or drinking products with grapefruit, as grapefruit is a strong CYP3A4 inhibitor and may increase the risk of adverse reactions from mavorixafor.

Active ingredient

Mavorixafor
Mavorixafor is an orally bioavailable CXC Chemokine Receptor 4 (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. Gain of function mutations in the CXCR4 receptor gene that occur in patients with WHIM syndrome lead to increased responsiveness to CXCL12 and retention of leukocytes in the bone marrow. Mavorixafor inhibits the response to CXCL12 in both wild-type and mutated CXCR4 variants associated with WHIM syndrome. Treatment with mavorixafor results in increased mobilization of neutrophils and lymphocytes from the bone marrow into peripheral circulation. Read more about Mavorixafor

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