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  5. Drug Trials Snapshots: XENPOZYME
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Drug Trials Snapshots: XENPOZYME

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 XENPOZYME Prescribing Information for all of 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).

XENPOZYME (olipudase alfa-rpcp)
(ZEN-poh-zyme)
Genzyme Corporation
Approval date: August 31, 2022


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

XENPOZYME is an enzyme replacement therapy that is used for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

ASMD is a rare disease where patients have decreased activity of the enzyme sphingomyelinase, which breaks down a fatty lipid called sphingomyelin. Sphingomyelin can build up in organs and tissues, especially the lungs, liver, and spleen, causing them to stop working.

How is this drug used?

XENPOZYME is given by a healthcare provider using a needle placed in a vein (known as an intravenous infusion) every two weeks.

Who participated in the clinical trials?

XENPOZYME’s safety and efficacy data were derived from three clinical trials: Trial 1 in adult patients (NCT02004691), Trial 2 in pediatric patients (NCT02292654), and Trial 3 a long-term trial in pediatric patients (NCT02004704). These trials enrolled a total of 30 adult and 8 pediatric patients with ASMD. The trials were conducted at 30 sites in 24 countries around the world, including the United States.

How were the trials designed?

XENPOZYME was evaluated in three clinical trials of 38 patients with ASMD. Trial 1 evaluated the benefits and side effects of XENPOZYME in adult patients. Trial 2 evaluated the benefit and side effects of XENPOZYME in pediatric patients. Trial 3 was an extension of Trial 2 that evaluated benefit and side effects of XENPOZYME in pediatric patients.

In Trial 1, adult patients received either XENPOZYME or a placebo intravenously once every 2 weeks for 52 weeks. Neither the patients nor the healthcare providers knew which treatment was being given until after Week 52. In Trial 2, pediatric patients received XENPOZYME intravenously once every 2 weeks for 64 weeks.

Trial 3 was an open label long term trial that continued to evaluate pediatric patients that had completed Trial 2.

The benefit of XENPOZYME was evaluated by assessing the diffusion capacity of the lung for carbon monoxide, spleen volume, liver volume and platelet count.


DEMOGRAPHICS SNAPSHOT

Figure 1. Baseline Demographics by Sex (Trials 1, 2, and 3)

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 16 (42%) male patients and 22 (68%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2. Baseline Demographics by Race (Trials 1, 2, and 3)

Pie chart summarizing how many White, Asian, and other patients were in the clinical trial. In total, 34 (90%) White patients, 2 (5%) Asian patients, and 2 (5%) Other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3. Baseline Demographics by Age (Trials 1, 2, and 3)

Pie chart summarizing how many patients by age were in the clinical trial. In total, 1 (3%) infant patient younger than 2 years of age, 7 (18%) child patients between 2 and 12 years of age, 29 (76%) adult patients between 18 and 65 years of age, and 1 (3%) elderly patient older than 65 years of age participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4. Baseline Demographics by Ethnicity (Trials 1, 2, and 3)

Pie chart summarizing how many Hispanic, Not Hispanic, and not reported patients were in the clinical trial. In total, 10 (26%) Hispanic or Latino patients, 27 (71%) Not Hispanic or Latino patients, and 1 (3%) not reported patients participated in the clinical trial.

What are the benefits of this drug?

XENPOZYME improved lung function and improved the size of the spleen in adult patients after 52 weeks of treatment. A similar effect was also seen in children after 64 weeks of treatment.

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

  • Sex: The number of patients in the trials was small; therefore, differences in how XENPOZYME worked among sexes could not be determined.
  • Race: The number of patients in the trials was small; therefore, differences in how XENPOZYME worked among races could not be determined.
  • Age: XENPOZYME worked similarly in all age groups studied.

What are the possible side effects?

XENPOZYME may cause serious side effects such as hypersensitivity reaction and infusion associated reactions because XENPOZYME is an enzyme replacement therapy.

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

  • Sex: The number of patients in the trials was small; therefore, differences in the occurrence of side effects among sexes could not be determined.
  • Race: The number of patients in the trials was small; therefore, differences in the occurrence of side effects among races could not be determined.
  • Age: Most of the patients were younger than 65 years of age. The occurrence of side effects was more frequent in patients younger than 18 years of age.

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 used in clinical trials 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.

PRESCRIBING INFORMATION

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