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Drug Trials Snapshots: VOYDEYA

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 VOYDEYA 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).

VOYDEYA (danicopan)
(voi-day-uh)
Alexion Pharmaceuticals
Approval date: March 29, 2024


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

VOYDEYA is a prescription drug that is a complement factor D inhibitor, indicated as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH)

How is this drug used?

VOYDEYA is an oral capsule that is taken three times daily.

Who participated in the clinical trials?

The FDA approved VOYDEYA based on evidence from one main clinical trial, ALXN2040-PNH-301, in 84 subjects with PNH and clinically significant EVH. Efficacy was based on a prespecified interim analysis when approximately 75% of the planned 84 subjects (63 subjects) reached the end of Treatment Period 1 (VOYDEYA [n=42] or placebo [n=21]). The trial was conducted in 15 countries including Spain, France, Italy, Poland, Japan, Brazil, Canada, Korea, Malaysia, Thailand, Czechia, Greece, Israel, United Kingdom, and the United States.

There were 9 subjects included in the ALXN2040-PNH-301 trial from the United States and 54 subjects were included from sites outside of the United States. The same trial was used to assess efficacy and safety.

How were the trials designed?

VOYDEYA was evaluated in one main clinical trial of subjects with PNH and clinically significant EVH. Study ALXN2040-PNH-301 was a multi-center, randomized, double-blind, placebo-controlled 24-week trial in adults with PNH with clinically significant EVH on treatment with a stable regimen of an anti-C5 treatment (either eculizumab or ravulizumab) for at least six months prior. The patients were randomized to VOYDEYA or placebo for 12 weeks in addition to background ravulizumab or eculizumab treatment. After Week 12, all patients received VOYDEYA in combination with their background ravulizumab or eculizumab treatment up to Week 24.


DEMOGRAPHICS SNAPSHOT

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

Figure 1. Baseline Demographics by Sex, Safety Population

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 32 (37%) male patients and 54 (63%) female patients participated in the clinical trial.

Source: Adapted from FDA review

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

Figure 2. Baseline Demographics by Race, Safety Population

Pie chart summarizing how many White, Asian, Black or African American, American Indian or Alaska Native, unknown, other, and not reported patients were in the clinical trial. In total, 42 (49%) White patients, 32 (37%) Asian patients, 2 (3%) Black or African American patients, 1 (1%) American Indian or Alaska Native patient, 1 (1%) unknown patient, 1 (1%) other patient, and 7 (8%) race not reported patients participated in the clinical trial.

Source: Adapted from FDA review

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

Figure 3. Baseline Demographics by Age, Safety Population

Pie chart summarizing how many patients by age were in the clinical trial. In total, 64 (74%) patients younger than 65 years of age, 13 (15%) patients between 65 and 75 years of age, and 9 (11%) patients 75 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 clinical trial used to evaluate the efficacy of VOYDEYA.

Figure 4. Baseline Demographics by Ethnicity, Safety Population

Pie chart summarizing how many Hispanic, not Hispanic, and not reported patients were in the clinical trial. In total, 7 (8%) Hispanic or Latino patients, 70 (81%) not Hispanic or Latino patients, and 9 (11%) ethnicity not reported patients participated in the clinical trial.

Source: Adapted from FDA review

What are the benefits of this drug?

One trial in adults with PNH and EVH despite previous treatment with a stable regimen of anti C5 treatment (either eculizumab or ravulizumab) for at least six months, demonstrated superiority of VOYDEYA in combination with ravulizumab or eculizumab compared to placebo in combination with ravulizumab or eculizumab in all efficacy measures with statistically significant results.

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

  • Sex: VOYDEYA worked similarly in males and females.
  • Race: The number of patients of races other than White was small; therefore, differences in how VOYDEYA worked among races could not be determined.
  • Age: VOYDEYA worked similarly in patients younger and older than 45 years of age.

What are the possible side effects?

VOYDEYA can cause hepatic enzyme increases, hyperlipidemia, and patients are to be monitored for PNH manifestations after VOYDEYA discontinuation for signs of hemolysis. The most common side effect of VOYDEYA was headache.

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 number of patients of races other than White was small; therefore, the occurrence of side effects of VOYDEYA could not be determined.
  • Age: The occurrence of side effects was similar in patients younger and older than 45 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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