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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.

How were the trials designed?

The efficacy and safety of RYSTIGGO for the treatment of gMG in adult patients who are anti-AChR antibody positive or anti-MuSK antibody positive was established in a multicenter, randomized, double-blind, placebo-controlled study (Study 1, NCT03971422). All patients had gMG defined as Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa and Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score of at least 3 (with at least 3 points from non-ocular symptoms) on stable dose of MG therapy including acetylcholine esterase inhibitors, steroids, or non-steroidal immunosuppressive therapies, either alone or in combination.

In Study 1, patients received RYSTIGGO equivalent to 7 mg/kg or 10 mg/kg, or placebo subcutaneous infusion every week for a period of six weeks (called a treatment cycle).

The primary efficacy outcome measure for Study 1 was the comparison of the change from baseline at Day 43 (after six weekly infusions of RYSTIGGO or placebo) in the MG-ADL total score between RYSTIGGO and placebo groups. 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

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

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

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

Source: Adapted from FDA Review

Who participated in the trials?

Table 1 summarizes the demographic data for patients enrolled in Study 1 used to evaluate the efficacy and safety of RYSTIGGO.

Table 1. Baseline Demographics Characteristics

Demographic Parameters	RYSTIGGO ≈7 mg/kg N=66	RYSTIGGO ≈10 mg/kg N=67	Placebo N=67	All N=200
Sex, n (%)
Female	39 (59)	35 (52)	47 (70)	121 (61)
Male	27 (41)	32 (48)	20 (30)	79 (39)
Age, years, n (%)
18 to <65	49 (74)	51 (76)	51 (76)	151 (75)
≥65	17 (26)	16 (24)	16 (24)	49 (25)
Race, n (%)
American Indian or Alaska Native	0	0	1 (1)	1 (1)
Asian	9 (14)	7 (10)	5 (7)	21 (10)
Black or African American	0	4 (6)	1 (1)	5 (2)
White	41 (62)	49 (73)	46 (69)	136 (68)
Not reported	16 (24)	7 (10)	14 (21)	37 (19)
Ethnicity, n (%)
Hispanic or Latino	5 (8)	3 (4)	5 (7)	13 (6)
Non-Hispanic or Latino	47 (71)	58 (87)	48 (72)	153 (77)
Not reported	14 (21)	6 (9)	14 (21)	34 (17)
Region, n (%)
Japan	5 (8)	4 (6)	4 (6)	13 (6)
United States	11 (17)	14 (21)	16 (24)	41 (21)
Rest of the world	50 (76)	49 (73)	47 (70)	146 (73)
BMI (kg/m2)
Mean (SD)	27 (7)	28 (6)	28 (6)	28 (6)
Median (min, max)	26 (14, 48)	27 (15, 45)	28 (14, 42)	27 (14, 48)

Source: Adapted from FDA Review
Abbreviations; BMI, body mass index; SD, standard deviation

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.

What are the benefits of this drug (results of trials used to assess efficacy)?

Table 2 summarizes the efficacy results for the patients who were evaluated in a clinical trial of RYSTIGGO. The primary outcome of the trial was a change from baseline to Day 43 (after six weekly infusions of RYSTIGGO) on the MG-ADL scale. The MG-ADL measures how patients feel about the effect of the disease on their daily activities. A total score ranges from 0 to 24. Higher scores represent greater severity of the disease.

The secondary outcome of the trial was a change from baseline to Day 43 in the Quantitative Myasthenia Gravis (QMG) total score. The QMG assesses muscle weakness. A total score ranges from 0 to 39. Higher scores indicate more severe weakness .

Table 2. Change From Baseline to Day 43 in MG-ADL and QMG Total Score in Adult Patients who are Anti-AChR or Anti-MuSK Antibody Positive in Study 1

Efficacy Endpoints	RYSTIGGO ≈7mg/kg N=66	RYSTIGGO ≈10mg/kg N=67	Placebo N=67
MG-ADL total score
LS mean (SE)	-3.4 (0.5)	-3.4 (0.5)	-0.8 (0.5)
Difference from placebo (95% CI)	-2.6 (-4.1, -1.2)	-2.6 (-4.0, -1.2)	-
p-value	<0.001	<0.001	-
QMG total score
LS mean (SE)	-5.4 (0.7)	-6.7 (0.7)	-1.9 (0.7)
Difference from placebo (95% CI)	-3.5 (-5.6, -1.6)	-4.8 (-6.8, -2.9)	-
p-value	<0.001	<0.001	-

Source: RYSTIGGO Prescribing Information
Abbreviations: AChR, acetylcholine receptor; CI, confidence interval; LS, least square; MG-ADL, Myasthenia Gravis Activities of Daily Living; MuSK, muscle-specific kinase; SE, standard error; QMG, Quantitative Myasthenia Gravis

Figure 5 shows the average change in the total MG-ADL score over time from the start of the treatment cycle in Study 1. The last dose of the treatment was given on Day 36 (arrows indicate timepoint at which treatment was given). The bottom curve shows the response in RYSTIGGO-treated patients.

Figure 5. Average Change in Total MG-ADL Score from the Beginning of Treatment Over Time in Study 1

Source: RYSTIGGO Prescribing Information
Note: Error bars represent ± standard error; arrows indicate timepoints at which treatment was given. Abbreviations: CfB, change from baseline; FV, final visit; MG-ADL, myasthenia gravis activities of daily living scale

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.

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

Table 3 summarizes the results of the subgroup analyses by age and sex. RYSTIGGO worked similarly in patients younger and older than 65 years of age, and in males and females. Differences in how well the drug worked among races could not be determined because of the small number of patients in some racial categories.

Table 3. Subgroup Analysis of Change from Baseline to Day 43 in MG-ADL in Study 1

Demographic Parameters	RYSTIGGO ≈7 mg/kg	RYSTIGGO ≈10 mg/kg	Placebo
Age group, years
18 to <65, N	48	49	47
LS mean (SE)	-3.57 (0.52)	-3.76 (0.54)	-0.99 (0.53)
Difference from placebo (95% CI)	-2.58 (-4.34, -1.17)	-2.77 (-4.34, -1.18)	-
≥65, N	17	16	15
LS mean (SE)	-1.51 (1.26)	-1.08 (1.22)	0.78 (1.13)
Difference from placebo (95% CI)	-2.29 (-7.26, 0.76)	-1.85 (-5.75, 1.58)	-
Sex
Female, N	38	34	42
LS mean (SE)	-3.60 (0.59)	-4.02 (0.59)	-0.89 (0.55)
Difference from placebo (95% CI)	-2.71 (-4.57, -1.17)	-3.13 (-4.99, -1.40)	-
Male, N	27	31	20
LS mean (SE)	-3.12 (0.91)	-2.85 (0.98)	-0.50 (1.04)
Difference from placebo (95% CI)	-2.62 (-5.30, 0.02)	-2.35 (-4.83, 0.20)	-

Source: Adapted from FDA Review
Abbreviations: CI, confidence interval; LS, least square; MG-ADL, myasthenia gravis activities of daily living scale; N, number of subjects with data in each arm; SE, standard error

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.

What are the possible side effects (results of trials used to assess safety)?

Table 4 summarizes the most common side effects of RYSTIGGO that occurred in Study 1.

Table 4. Adverse Reactions in at Least 5% of Patients Treated with RYSTIGGO and More Frequently Than in Patients Who Received Placebo in Study 1

Adverse Reaction	RYSTIGGO N=133 %	Placebo N=67 %
Headache¹	44	19
Any infection	23	19
Diarrhea	20	13
Pyrexia (fever)	17	2
Hypersensitivity reactions	11	5
Nausea	10	8
Administration site reactions	8	3
Abdominal pain	8	6
Arthralgia (joint pain)	7	3

Source: RYSTIGGO Prescribing Information
¹ Headache includes migraine

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.

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

Table 5 summarizes the results of the subgroup analysis by age, sex, and race. The majority of patients in Study 1 were female. Differences in side effects among some age and racial groups could not be determined due to small number of patients enrolled in the study.

Table 5. Subgroup Analysis of Side Effects Occurring in at Least 5% of Patients Treated with RYSTIGGO and More Frequently Than Placebo in Study 1

Demographic Parameters	RYSTIGGO N=133 n/Ns (%)	Placebo N=67 n/Ns (%)
Age group, years
≤18	0	1/1 (100)
19 to <65	69/100 (69)	19/50 (38)
≥65	16/33 (48)	8/16 (50)
Sex
Female	53/74 (72)	22/47 (47)
Male	32/59 (54)	6/20 (30)
Race
Asian	12/16 (75)	2/5 (40)
Black or African American	2/4 (50)	0/1 (0)
Native Hawaiian or other Pacific Islander	0	0/1 (0)
White	58/90 (64)	18/46 (39)
Other	13/23 (56)	8/14 (57)

Source: Adapted from FDA Review
Abbreviation: N, number of patients in the safety population; n, number of patients with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific arm

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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