Xolremdi: A New Treatment for WHIM Syndrome Approved on April 26, 2024
What is it prescribed for?
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 lymphocytes.
What is the name of the drug and what does it do?
Xolremdi (pronounced zohl-REM-dee), (generic name: mavorixafor), is a medication indicated for patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections, and myelokathexis).
It helps increase the number of circulating mature neutrophils and lymphocytes, improving immune function.
How does it work?
Xolremdi is a CXC chemokine receptor 4 (CXCR4) antagonist. It works by blocking the CXCR4 receptor, which is involved in the retention of white blood cells in the bone marrow.
By inhibiting this receptor, Xolremdi mobilizes neutrophils and lymphocytes from the bone marrow into the bloodstream, enhancing the body’s ability to fight infections and improving overall immune function.
What did the research discover?
Clinical trials demonstrated that Xolremdi significantly increases the number of circulating neutrophils and lymphocytes in patients with WHIM syndrome.
In the trials, patients treated with Xolremdi showed a substantial improvement in their absolute neutrophil count (ANC) and absolute lymphocyte count (ALC).
The trials also reported a reduction in infection rates and an improvement in the overall health status of the patients.
What are some of the side effects?
- Thrombocytopenia
- Pityriasis
- Rash
- Rhinitis
- Epistaxis
- Vomiting
- Dizziness
What are the dosage recommendations and how is it prescribed?
Xolremdi is administered orally. The recommended dosage is based on the patient's weight:
- For patients weighing more than 50 kg: 400 mg once daily.
- For patients weighing 50 kg or less: 300 mg once daily.
Xolremdi should be taken on an empty stomach after an overnight fast and at least 30 minutes before food. The capsules should be swallowed whole without breaking, chewing, or opening them. If a dose is missed, the next dose should be taken as scheduled, without doubling the dose.
Source:
- X4 Pharmaceuticals, Inc. Xolremdi (mavorixafor) capsules, for oral use. Prescribing Information. 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218709s000lbl.pdf