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  5. Drug Trials Snapshots: RYSTIGGO
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Drug Trials Snapshots: RYSTIGGO

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 RYSTIGGO 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).

RYSTIGGO (rozanolixizumab-noli)
ris-TIG-oh
UCB, Inc.
Original Approval date
: June 26, 2023


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

RYSTIGGO is a drug for the treatment of generalized myasthenia gravis (gMG) in adult patients. It is used by patients whose blood has antibodies against the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK).

Myasthenia gravis is a rare disease that causes weakness in muscles, especially those muscles that control the eyes, face, neck, mouth, swallowing, breathing, and limbs.

How is this drug used?

A healthcare provider injects RYSTIGGO underneath the skin of the lower right or left part of the abdomen through an infusion pump. This is known as subcutaneous infusion.

RYSTIGGO is given once weekly for six weeks (called a treatment cycle). If a patient’s symptoms return or worsen, then the patient may be eligible to receive another treatment cycle at least 63 days after the start of the previous treatment cycle.

The amount of RYSTIGGO used depends on the patient’s weight.

Who participated in the clinical trials?

The FDA approved RYSTIGGO based on evidence from Study 1 in 200 adult patients with gMG whose blood had antibodies against AChR or MuSK. The study was conducted at 81 sites in 15 countries in Canada, Czech Republic, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Poland, Russian Federation, Serbia, Spain, Taiwan, and the United States.

How were the trials designed?

The benefit and side effects of RYSTIGGO were established in one multicenter, randomized, double-blind, placebo-controlled trial (Study 1). The study evaluated RYSTIGGO for the treatment of gMG in adult patients whose blood had antibodies against AChR or MuSK.

Patients were randomized to receive weight-tiered doses of RYSTIGO equivalent to 7mg/kg or 10 mg/kg, or placebo subcutaneous infusion once weekly for six weeks (called a treatment cycle).

The primary efficacy endpoint was the comparison of the change from baseline after 43 days of treatment on the Myasthenia Gravis Activities of Daily Living (MG-ADL) total score between patients treated with RYSTIGGO and patients who received placebo, during one treatment cycle in Study 1. The MG-ADL is a scale reported by patients that assesses the impact of gMG on daily function.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many females and males were enrolled in Study 1 used to evaluate the efficacy and safety of RYSTIGGO.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 79 (39%) male patients and 121 (61%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race enrolled in the Study 1 used to evaluate the efficacy and safety of RYSTIGGO.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many White, Black or African American, Asian, American Indian or Alaska Native, and not reported patients were in the clinical trial. In total, 136 (68%) White patients, 5 (2%) Black or African American patients, 21 (10%) Asian patients, 1 (1%) American Indian or Alaska Native, and 37 (19%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes how many patients by age enrolled in the Study 1 used to evaluate the efficacy and safety of RYSTIGGO.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 151 (75%) patients between 18 and 65 years of age and 49 (25%) 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 enrolled in the Study 1 used to evaluate the efficacy and safety of RYSTIGGO.

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, 13 (6%) Hispanic or Latino patients, 153 (77%) not Hispanic or Latino patients, and 34 (17%) not reported patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

The patients who received RYSTIGGO experienced less weakness affecting their activities of daily living compared to those receiving the placebo infusions.

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

  • Sex: The majority of patients in the trial were female because myasthenia gravis affects females more frequently than males. RYSTIGGO worked similarly in males and females.
  • Race: The number of patients of races other than White was small; therefore, differences in how RYSTIGGO worked among races could not be determined.
  • Age: RYSTIGGO worked similarly in patients younger and older than 65 years of age.

What are the possible side effects?

RYSTIGGO may increase the risk for infections. RYSTIGGO may cause drug-induced aseptic meningitis. RYSTIGGO is associated with hypersensitivity reactions including angioedema and rash.

The most common side effects of RYSTIGGO are headache, infection, diarrhea, pyrexia (fever), hypersensitivity reactions, nausea, administration site reactions, abdominal pain, and joint pain.

Were there any differences in side effects of the clinical trials 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, differences in side effects among races could not be determined.
  • Age: The number of patients aged 65 years and older was small; therefore, the differences in side effects among age groups 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 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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