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  5. Drug Trials Snapshots: ELFABRIO
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Drug Trials Snapshots: ELFABRIO

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 ELFABRIO 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).

ELFABRIO (pegunigalsidase alfa-iwx) 
Chiesi Farmaceutici S.p.A.
Original Approval date:
May 9, 2023
 


DRUG TRIALS SNAPSHOT SUMMARY

What is the drug for?

ELFABRIO is an enzyme replacement therapy that is used for the treatment of Fabry disease in adult patients. Fabry disease is an inherited disorder that occurs when the enzyme alpha-galactosidase-A is deficient, affecting the breakdown of a type of fat called globotriaosylceramide (Gb3) in various tissues of the body such as the kidney, heart, brain, nerves, and digestive system.

How is this drug used?

ELFABRIO is administered 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?

ELFABRIO’s safety and efficacy data came from two clinical trials. Trial 1, evaluating efficacy, enrolled 16 Fabry disease patients who had never previously received enzyme replacement therapy or who had not received enzyme replacement therapy for more than 26 weeks prior to study entry. Trial 2, evaluating safety, enrolled 77 Fabry disease patients who had previously been treated with enzyme replacement therapy (agalsidase beta). Trial 1 was conducted at 13 study sites from 6 countries; Trial 2 was conducted at 28 study sites from 12 countries.

How were the trials designed? 

Trial 1, designed to identify optimal dose and evaluate efficacy, enrolled 16 Fabry disease patients who had never previously received enzyme replacement therapy or who had not received enzyme replacement therapy for more than 26 weeks prior to study entry. Trial 2, designed the evaluate safety, enrolled 77 Fabry disease patients who had previously been treated with enzyme replacement therapy (agalsidase beta).

In Trial 1, patients received ELFABRIO at doses of either 0.2 mg/kg, 1.0 mg/kg, or 2.0 mg/kg intravenously once every two weeks. The approved dose is 1.0 mg/kg. The efficacy and safety of the 0.2 mg/kg and 2.0 mg/kg have not been established and are not approved or recommended. The study endpoint was assessed six months following treatment initiation. There was no comparator group in Trial 1. In Trial 2, patients received ELFABRIO intravenously once every two weeks for 24 months. The comparator group in Trial 2 was agalsidase beta.

The benefit of ELFABRIO in Trial 1 was evaluated by assessing the amount of Gb3 deposition in the kidneys assessed by renal biopsy. Trial 2 evaluated the effects of ELFABRIO on safety and tolerability and also on estimated glomerular filtration rate (a measure of kidney function).

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the combined clinical trials used to evaluate the efficacy and safety of ELFABRIO.

Figure 1. Baseline Demographics by Sex, Combined Efficacy and Safety Populations

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 56 (60%) male patients and 37 (40%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race enrolled in the combined clinical trials used to evaluate the efficacy and safety of ELFABRIO.

Figure 2. Baseline Demographics by Race, Combined Efficacy and Safety Populations

Pie chart summarizing how many White, Black or African American, Asian or Pacific Islander, and other patients were in the clinical trial. In total, 84 (90%) White patients, 6 (6%) Black or African American patients, 2 (2%) Asian or Pacific Islander patients, and 1 (1%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes how many patients by age enrolled in the combined clinical trials used to evaluate the efficacy and safety of ELFABRIO.

Figure 3. Baseline Demographics by Age, Combined Efficacy and Safety Populations

Pie chart summarizing how many patients by age were in the clinical trial. In total, 1 (17%) patient younger than 18 years of age, and92 (99%) patients between 18 and 65 years of age participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes how many patients by ethnicity enrolled in the combined clinical trials used to evaluate the efficacy and safety of ELFABRIO.

Figure 4. Baseline Demographics by Ethnicity, Combined Efficacy and Safety Populations

Alt-Tag: Pie chart summarizing how many Hispanic, not Hispanic, and other patients were in the clinical trial. In total, 5 (5%) Hispanic or Latino patients, 88 (95%) not Hispanic or Latino patients, and 50 (9%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug? 

ELFABRIO reduced the amount of Gb3 deposited in kidney tissue of adult Fabry patients after six months of treatment. Reduction of Gb3 deposits in the kidney, for example, is expected to slow the rate of progression of kidney disease.

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

The number of patients in the trial was too small to determine if there were differences in how well the drug worked based on sex, race, or age.

What are the possible side effects?

ELFABRIO may cause serious side effects such as hypersensitivity reactions and infusion-associated reactions.

The most common side effects are infusion-associated reactions, nasal and throat irritation (nasopharyngitis), headache, diarrhea, fatigue, nausea, back pain, pain in arms or legs, joint pain (arthralgia), and sinus infection.

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

  • Sex: The number of participants enrolled in the trials were too small to determine if side effects differed between males and females.
  • Race: The number of patients of races other than White was small; therefore, differences side effects among races could not be determined.
  • Age: No patients older than 65 years of age were included, therefore, the differences in occurrence of side effects been patients younger and older than 65 years of age could not be determined. 

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.

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