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Drug Trials Snapshots: JOENJA

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 JOENJA 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).

JOENJA (leniolisib)
jo en' jah
Pharming Technologies BV
Original Approval date
: March 24, 2023


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

JOENJA is a kinase inhibitor that is used to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older who weigh at least 45 kg.

APDS is an inherited disease that impacts the immune system. APDS often causes a decrease in T and B cells that fight infection as well as an increase in lymph node size.

How is this drug used?

JOENJA is a tablet taken by mouth every 12 hours.

Who participated in the clinical trials?

The FDA approved JOENJA based on evidence from a clinical trial of 31 patients with APDS. The trial was conducted at 10 sites in 9 countries in Italy, the United States, United Kingdom, Czech Republic, Netherlands, Ireland, Belarus, Russia, and Germany.

How were the trials designed?

JOENJA was evaluated in one placebo-controlled clinical trial of 31 patients with APDS. Patients were randomized 2:1 to receive either JOENJA 70 mg (N=21) or placebo (N=10) twice a day for 12 weeks. The coprimary efficacy endpoints were a reduction in the size of lymph nodes and the increase in percentage of naïve B cells. Neither the patients nor the investigators knew which treatment was being given until after the trial was completed.

Investigators measured the size of lymph nodes before the first dose of JOENJA or placebo and after the end of the study by imaging. Blood samples were taken from patients during the 12 week treatment period to record the number of naïve B cells in the patient’s blood.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many males and females were enrolled in the clinical trial used to evaluate the efficacy of JOENJA.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 15 (48%) male patients and 16 (52%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes the percentage of patients by race enrolled in the clinical trial used to evaluate the efficacy of JOENJA.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many White, Black or African American, Asian, and other patients were in the clinical trial. In total, 25 (81%) White patients, 2 (6%) Black or African American patients, 2 (6%) Asian patients, and 2 (6%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes the percentage of patients by age enrolled in the clinical trial used to evaluate the efficacy of JOENJA.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 12 (39%) patients between 12 and 18 years of age, 17 (55%) patients between 18 and 45 years of age, and 2 (6%) patients between 45 and 65 years of age participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes the percentage of patients by ethnicity enrolled in the clinical trial used to evaluate the efficacy of JOENJA.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many Hispanic, not Hispanic, and not reported patients were in the clinical trial. In total, 1 (3%) Hispanic or Latino patient, 21 (68%) not Hispanic or Latino patients, and 9 (29%) not reported patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

In patients with APDS, JOENJA increased the percentage of B cells important in fighting infections and decreased the size of lymph nodes.

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

  • Sex: JOENJA worked similarly in males and females.
  • Race: Most of the patients were White. Differences in how well the drug worked among races could not be determined because of the small number of patients in other races.
  • Age: All patients were between 12 to 54 years of age; therefore, differences in how well the drug worked among older age groups could not be determined. JOENJA worked similarly between patients younger and older than 18 years of age.

What are the possible side effects?

The most common adverse reactions (>10%) were headache, sinusitis, and atopic dermatitis. Adverse reactions reported by two or more patients treated with JOENJA and more frequently than placebo are summarized in Table 5.

Were there any differences in side effects of the clinical trials among sex, race, and age?

  • Sex: The occurrence of side effects was slightly higher in females than in males. There were no major differences in the patterns or types of side effects between females and males. Because of limited data, this difference may be due to chance.
  • Race: Most of the patients were White. Differences in the occurrence of side effects among races could not be determined because of the small number of patients in other races.
  • Age: All patients were 12 to 54 years of age; therefore, differences in the occurrence of side effects among older age groups could not be determined. The occurrence of side effects was similar between patients younger and older 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 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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