Xolremdi FDA Approval History
Last updated by Judith Stewart, BPharm on April 29, 2024.
FDA Approved: Yes (First approved April 26, 2024)
Brand name: Xolremdi
Generic name: mavorixafor
Dosage form: Capsules
Company: X4 Pharmaceuticals
Treatment for: WHIM Syndrome
Xolremdi (mavorixafor) is a CXC chemokine receptor 4 (CXCR4) antagonist indicated in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.
- WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. People with WHIM syndrome characteristically have neutropenia and lymphopenia, and experience frequent, recurrent infections with a high risk of lung disease, refractory warts from underlying human papillomavirus (HPV) infection, limited antibody production due to low levels of immunoglobulin, and an increased risk of developing certain types of cancer.
- Xolremdi works in the treatment of WHIM syndrome by increasing mobilization and trafficking of white blood cells from the bone marrow into the bloodstream so that the body can better fight infections.
- FDA approval of Xolremdi was based on results of the 52-week, placebo-controlled 4WHIM Phase 3 clinical trial which demonstrated increased time above threshold for absolute neutrophil count and absolute lymphocyte count versus placebo. Results also showed an approximate 40% reduction in total infection score (weighted by infection severity) and a 60% reduction in the annualized infection rate in Xolremdi-treated patients compared with placebo-treated patients. There was no difference in total wart change scores between the Xolremdi and placebo treatment arms.
- Xolremdi capsules are administered orally, once daily on an empty stomach after an overnight fast, and at least 30 minutes before food.
- Warnings and precautions associated with Xolremdi include fetal harm and QTc interval prolongation.
- Common adverse reactions include thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.
- Mavorixafor is also being studied in combination with ibrutinib for the treatment of Waldenström’s macroglobulinemia.
Development timeline for Xolremdi
Further information
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