XOLREMDI (mavorixafor)

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. Mavorixafor demonstrates nonlinear pharmacokinetics with greater than dose-proportional increases in C max and AUC 0-24 over a dose range of 50 mg (0.125 times the recommended dosage) to 400 mg. Mavorixafor steady s...

The efficacy of XOLREMDI in patients aged 12 and older with WHIM syndrome was demonstrated in the 52-week, randomized, double-blind, placebo-controlled portion of Study 1 [ NCT03995108 ]. Enrolled patients had a genotype-confirmed variant of CXCR4 consistent with WHIM syndrome, and a confirmed absolute neutrophil count (ANC) ≤400 cells/µL. Patients were permitted to continue (but not initiate) immunoglobulin therapy at the same dose. Use of other CXCR4 antagonists was not permitted. Baseline patient demographics are shown in Table 2.

Table 2Baseline Demographic and Disease Characteristics in...

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 Cli...

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. ( 4 , 7.2 )

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 admin...

7 DRUG INTERACTIONS Strong CYP3A4 inhibitors: Reduce XOLREMDI daily dosage. ( 2.2 , 7.1 ) P-gp inhibitors or moderate CYP3A4 inhibitors: Monitor more frequently for XOLREMDI adverse reactions and reduce XOLREMDI daily dosage if necessary. ( 7.1 ) Strong CYP3A4 Inducers: Avoid concomitant use. ( 7.1 ) CYP3A4 or P-gp substrates: Monitor more frequently for substrate adverse reactions unless otherwis...