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Drug Trials Snapshots: XOLREMDI

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the XOLREMDI Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

XOLREMDI (mavorixafor) 
zol-REM-dee
X4 Pharmaceuticals, Inc.
Original Approval date: April 26, 2024


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

XOLREMDI is a prescription drug that increases the number of white blood cells released from the bone marrow into the blood. XOLREMDI is indicated for the treatment of adults and adolescents over the age of 12 years with WHIM (Warts, Hypogammaglobulinemia, Infection, and Myelokathexis) syndrome.

How is this drug used?

XOLREMDI is a capsule taken by mouth once a day.

Who participated in the clinical trials?

The FDA approved XOLREMDI based on evidence from a clinical trial (NCT03995108) of 31 patients with WHIM syndrome. The trial was conducted at 19 sites in 12 countries in North America, Europe, Australia, and Asia. Of the 31 patients, 6 patients were from trial sites in the United States.

The benefits and side effects of XOLREMDI were evaluated in the same single clinical trial.

How were the trials designed?

XOLREMDI was evaluated in one clinical trial of 31 patients with WHIM syndrome.

The trial was a randomized, double-blind, placebo-controlled trial in patients aged 12 years and older with WHIM syndrome. The patients were randomized to receive either placebo or XOLREMDI once daily for 52 weeks. The efficacy of XOLREMDI in the treatment of patients with WHIM syndrome was based on improvement in absolute neutrophil counts (ANC), improvement in absolute lymphocyte counts (ALC), and a reduction in infections.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the trial used to evaluate the efficacy of XOLREMDI.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 13 (42%) male patients and 18 (58%) female patients participated in the clinical trial.
Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race were enrolled in the trial used to evaluate the efficacy of XOLREMDI.

Figure 2. Baseline Demographics by Race 

Pie chart summarizing how many White, Asian, and other patients were in the clinical trial. In total, 29 (94%) White patients, 1 (3%) Asian patient, and 1 (3%) other patient participated in the clinical trial.
Source: Adapted from FDA Review

Figure 3 summarizes how many patients by age were enrolled in the trial used to evaluate the efficacy of XOLREMDI.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 15 (48%) patients younger than 18 years of age, 14 (45%) patients between 18 and 65 years of age, and 2 (7%) patients 65 years of age and older participated in the clinical trial.
Source: Adapted from FDA Review

Figure 4 summarizes how many patients by ethnicity were enrolled in the trial used to evaluate the efficacy of XOLREMDI.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many Hispanic and not Hispanic patients were in the clinical trial. In total, 1 (3%) Hispanic or Latino patient and 30 (97%) not Hispanic or Latino patients participated in the clinical trial.
Source: Adapted from FDA Review

What are the benefits of this drug? 

Compared to patients who had received placebo, patients who received XOLREMDI had higher numbers of white blood cells and fewer infections.

What are the possible side effects?

The most common side effects were thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

XOLREMDI may cause clinically significant QTc interval prolongation.

XOLREMDI is expected to cause fetal harm. Females of reproductive potential should use effective contraception and XOLREMDI should not be prescribed during pregnancy.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age? 

  • Sex: The effect of XOLREMDI was similar for females and males.
  • Race: The number of patients of races other than White was small; therefore, differences in how XOLREMDI worked among races could not be determined.
  • Age: The number of patients older than 65 years of age was small; therefore, differences in how XOLREMDI worked between patients younger and older than 65 years of age could not be determined. The effect of XOLREMDI was similar for patients younger and older than 18 years of age.

Were there any differences in side effects among sex, race, and age?

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The majority of patients in the trial were White. Differences in the occurrence of side effects could not be determined because of the small number of patients in other races.
  • Age: The majority of patients in the trial were younger than 65 years old. Differences in the occurrence of side effects in patients 65 years of age or older could not be determined because of the small number of patients.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

LINK TO DRUG PACKAGE INSERT

 

 
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