EFFICACY

Rapid efficacy in action for a broad population of adults with gMG1,2*

In a clinical study, RYSTIGGO demonstrated statistically significant improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs -0.8; P<0.001), with improvements observed as early as Week 1.1

The clinical study included adults who were anti-AChR Ab+ and anti-MuSK Ab+, and who had MGFA Class II-IVa disease, an MG-ADL score of ≥3 (with ≥3 points from non-ocular symptoms), serum IgG levels ≥5.5 g/L, and who were on a stable dose of MG therapy prior to screening.1

Rapid efficacy in action for a broad population of adults with gMG1,2*

In a clinical study, RYSTIGGO demonstrated statistically significant improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs -0.8; P<0.001), with improvements observed as early as Week 1.1

The clinical study included adults who were anti-AChR Ab+ and anti-MuSK Ab+, and who had MGFA Class II-IVa disease, an MG-ADL score of ≥3 (with ≥3 points from non-ocular symptoms), serum IgG levels ≥5.5 g/L, and who were on a stable dose of MG therapy prior to screening.1

PRIMARY ENDPOINT: MG-ADL

Adults taking RYSTIGGO experienced rapid, clinically meaningful, and statistically significant improvements in activities of daily living in a 6-week treatment cycle1,2†

Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+1-3‡

Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+.
Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+.

Clinically meaningful was established as a ≥2.0-point improvement in MG-ADL total score.2

During the treatment period, RYSTIGGO or placebo were administered subcutaneously once a week for 6 weeks.1

Treatment initiation on Day 1.2

Adults taking RYSTIGGO achieved maximum efficacy at Week 6 (Day 43).1

The efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG was established in an up to 18-week, multicenter, randomized, double-blind, placebo-controlled study. In the study, 200 patients were randomized 1:1:1 to receive weight-tiered doses of RYSTIGGO (n=133), either 7 mg/kg of RYSTIGGO (n=66) or 10 mg/kg of RYSTIGGO (n=67), or placebo (n=67). The study included a 4-week screening period and a 6-week treatment period followed by 8 weeks of observation.1

GO TO THE STUDY DESIGN

MG-ADL OUTCOME MEASURE

MG-ADL assesses the impact of gMG on daily functions of 8 symptoms on a scale of 0-3, with total scores ranging from 0-24. Higher scores are interpreted as greater impairments. An improvement of ≥2.0 points was established as clinically meaningful.1,2

Measures include4:

  • Voice/speech problems
  • Chewing
  • Swallowing
  • Breathing
  • Brushing teeth and/or combing hair
  • Rising from a chair
  • Diplopia
  • Eyelid droop
MG-ADL Guide

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SECONDARY ENDPOINT: MG-ADL Responder Rate

Proportion of MG-ADL responders from baseline at Week 6 (Day 43)2

MG-ADL responders: ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43)2

MG-ADL >2x more responders vs placebo.
MG-ADL >2x more responders vs placebo.

Study limitations: MG-ADL responder rate was a prespecified secondary endpoint not controlled for multiplicity; therefore, data should be interpreted with caution and conclusions cannot be drawn.

QMG and MGC responders were established as adults with a ≥3.0-point improvement in QMG and MGC score from baseline at Week 6 (Day 43).2

QMG responder rates from baseline at Week 6 (Day 43) were 54.7% (n=35/64) in the RYSTIGGO 7 mg/kg group and 72.6% (n=45/62) in the RYSTIGGO 10 mg/kg group compared to 39.1% (n=25/64) in the placebo group. MGC responder rates from baseline at Week 6 (Day 43) were 60.9% (n=39/64) in the RYSTIGGO 7 mg/kg group and 74.2% (n=46/62) in the RYSTIGGO 10 mg/kg group compared to 40.6% (n=26/64) in the placebo group.2

QMG OUTCOME MEASURE

QMG is a physician assessment scoring system that quantifies disease severity. Measures are assessed on a scale of 0-3, with total scores ranging from 0-39. An improvement of ≥3.0 points was established as clinically meaningful.1,2,5

Measures include5:

  • Ptosis
  • Facial muscle weakness
  • Dysarthria
  • Grip strength
  • Neck flexion endurance
  • Diplopia
  • Difficulty swallowing 4 oz of water
  • Percent predicted forced vital capacity
  • Arm and leg endurance

MGC OUTCOME MEASURE

MGC is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0-50. Higher scores are interpreted as greater impairments. An improvement of ≥3.0 points was established as clinically meaningful.2,6,7 

Measures include6:

  • Ptosis
  • Eye closure
  • Chewing
  • Breathing
  • Shoulder abduction
  • Diplopia
  • Talking
  • Swallowing
  • Neck flexion or extension
  • Hip flexion

OTHER MG-ADL EFFICACY ENDPOINT

Minimal Symptom Expression (MSE) rates observed during the pivotal study2

Other efficacy endpoint: Proportion of adults achieving MSE during treatment and observation in the pivotal study2

Adults who reached MSE achieved an MG-ADL total score of 0 to 1 at any time up to and including Week 6 (Day 43) of the pivotal study.
Patients who reached MSE achieved an MG-ADL total score of 0 to 1 at any time up to and including Week 6 (Day 43) of the pivotal study.

MSE was an exploratory endpoint and not controlled for multiplicity. Therefore, data should be interpreted with caution and conclusions cannot be drawn.

Watch a video featuring Dr. Christyn Edmundson, an expert in gMG, discussing the MG-ADL results from the RYSTIGGO pivotal study.
Watch a video featuring Dr. Christyn Edmundson, an expert in gMG, discussing the MG-ADL results from the RYSTIGGO pivotal study.

Review the RYSTIGGO MG-ADL results and MSE rates with a gMG expert

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Ab+=antibody positive; AChR=acetylcholine receptor; CI=confidence interval; gMG=generalized myasthenia gravis; IgG=immunoglobulin G; LS=least squares; MG‑‍ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGC=Myasthenia Gravis Composite; MGFA=Myasthenia Gravis Foundation of America; MuSK=muscle-specific tyrosine kinase; QMG=Quantitative Myasthenia Gravis; SE=standard error.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7
  3. Data on file. UCB Inc., Smyrna, GA.
  4. MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/ADL.pdf
  5. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/QMG.pdf
  6. MG composite scale. Myasthenia Gravis Foundation of America. 2012. Accessed February 27, 2025. https://myasthenia.org/‌Portals/‌0/‌MG%20composite%20score.pdf
  7. Regnault A, Morel T, de la Loge C, et al. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms PRO. Neurol Ther. 2023;12(5):1573-1590. doi:10.1007/s40120-023-00464-x

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SECONDARY ENDPOINTS

The efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG was established in an up to 18-week, multicenter, randomized, double-blind, placebo-controlled study. In the study, 200 patients were randomized 1:1:1 to receive weight-tiered doses of RYSTIGGO (n=133), either 7 mg/kg of RYSTIGGO (n=66) or 10 mg/kg of RYSTIGGO (n=67), or placebo (n=67).1

GO TO THE STUDY DESIGN 

QUANTITATIVE MYASTHENIA GRAVIS (QMG)

Adults taking RYSTIGGO experienced statistically significant improvements in QMG score vs placebo1

Secondary endpoint: Mean change in QMG score from baseline to Week 6 (Day 43)1

Quantitative Myasthenia Gravis (QMG) Score clinically meaningful improvement vs placebo.
Quantitative Myasthenia Gravis (QMG) Score clinically meaningful improvement vs placebo.

Improvements of ≥3.0 points were established as clinically meaningful.2

QMG OUTCOME MEASURE

QMG is a physician assessment scoring system that quantifies disease severity. Measures are assessed on a scale of 0-3, with total scores ranging from 0-39. An improvement of ≥3.0 points was established as clinically meaningful.1-3

Measures include3:

  • Ptosis
  • Facial muscle weakness
  • Dysarthria
  • Grip strength
  • Neck flexion endurance
  • Diplopia
  • Difficulty swallowing 4 oz of water
  • Percent predicted forced vital capacity
  • Arm and leg endurance

MYASTHENIA GRAVIS COMPOSITE (MGC)

Adults taking RYSTIGGO experienced statistically significant improvements in MGC score vs placebo2,4

Secondary endpoint: Mean change in MGC score from baseline to Week 6 (Day 43)2,4

Myasthenia Gravis Composite (MGC) Score clinically meaningful improvement vs placebo.
Myasthenia Gravis Composite (MGC) Score clinically meaningful improvement vs placebo.

Improvements of ≥3.0 points were established as clinically meaningful.2

MGC OUTCOME MEASURE

MGC is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0-50. Higher scores are interpreted as greater impairments. An improvement of ≥3.0 points was established as clinically meaningful.2,5,6 

Measures include5:

  • Ptosis
  • Eye closure
  • Chewing
  • Breathing
  • Shoulder abduction
  • Diplopia
  • Talking
  • Swallowing
  • Neck flexion or extension
  • Hip flexion

MYASTHENIA GRAVIS SYMPTOMS PATIENT-REPORTED OUTCOME (MG SYMPTOMS PRO)

MG Symptoms PRO evaluated gMG symptoms across 3 domains2:

MG SYMPTOMS PRO OUTCOME MEASURE

MG Symptoms PRO is a novel outcome measure developed by UCB and used in the RYSTIGGO clinical trials. It complements established clinical outcome measures and is the first outcome measure to include a standalone assessment of physical fatigue.2,6,7

MG Symptoms PRO is a patient assessment evaluating the symptom severity of myasthenia gravis. Symptoms are measured over a 7-day period. A score ranging from 0-100 is calculated for each domain, with a higher score indicating greater symptom severity.6

MG Symptoms PRO evaluates2,6:

Muscle weakness fatigability
A scale that measures muscle fatigability by assessing use-induced reduction in the ability of the following muscles to function:

  • Proximal
  • Ocular
  • Bulbar
  • Respiratory

Physical fatigue
A scale that measures the symptoms/manifestations of physical fatigue, including:

  • Body and limb muscle weakness
  • Heaviness
  • Lack of energy and strength

Bulbar muscle weakness
A scale that measures the symptoms/manifestations associated with bulbar muscle weakness, including:

  • Facial/mouth drooping
  • Speech and voice problems
  • Difficulties with chewing and swallowing
  • Liquid control in mouth
Video features Dr. Christyn Edmundson, an expert in gMG, discussing the secondary endpoint results, including MG Symptoms PRO, from the RYSTIGGO pivotal study.
Video features Dr. Christyn Edmundson, an expert in gMG, discussing the secondary endpoint results, including MG Symptoms PRO, from the RYSTIGGO pivotal study.

Watch a leading expert in gMG review the RYSTIGGO secondary endpoint results

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OTHER ENDPOINT

Reduction in immunoglobulin G (IgG) levels1

A reduction in total IgG serum concentrations was observed in adults in both RYSTIGGO treatment groups. Decreases in AChR autoantibody and MuSK autoantibody levels followed a similar pattern.1

Other endpoint: Mean % change from baseline to Week 6 (Day 43) in total IgG level4

Reduction in circulating IgG.
Reduction in circulating IgG.

Study limitations: The pharmacological effect of RYSTIGGO was assessed by measuring the decrease in serum IgG levels; the clinical significance of this data has not been established.

§Treatment initiation on Day 1.4

Ab+=antibody positive; AChR=acetylcholine receptor; CI=confidence interval; gMG=generalized myasthenia gravis; IgG=immunoglobulin G; MG-ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGFA=Myasthenia Gravis Foundation of America; MGSPRO=Myasthenia Gravis Symptoms Patient-Reported Outcome; MuSK=muscle-specific tyrosine kinase; SE=standard error.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7
  3. QMG form. Myasthenia Gravis Foundation of America. 1997. February 27, 2025. https://myasthenia.org/Portals/0/QMG.pdf
  4. Data on file. UCB, Inc., Smyrna, GA.
  5. MG composite scale. Myasthenia Gravis Foundation of America. 2012. February 27, 2025. https://myasthenia.org/Portals/0/MG%20composite%20score.pdf
  6. Regnault A, Morel T, de la Loge C, et al. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms PRO. Neurol Ther. 2023;12(5):1573-1590. doi:10.1007/s40120-023-00464-x
  7. Cleanthous S, Mork AC, Regnault A, et al. Development of the Myasthenia Gravis (MG) Symptoms PRO: a case study of a patient-centred outcome measure in rare disease. Orphanet J Rare Dis. 2021;16(1):1-14. doi:10.1186/s13023-021-02064-0

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EXTENSION STUDIES

The long-term safety and efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG was established in two Phase 3 extension studies, MG0004 and MG0007. Extension study results shown here are part of a pooled data analysis.3

GO TO THE STUDY DESIGN 

The primary endpoints in the RYSTIGGO extension studies evaluated the proportion of patients experiencing treatment-emergent adverse events (TEAEs). The most common TEAEs (>10%) reported in adults receiving either dose of RYSTIGGO in the extension studies were headache, diarrhea, COVID-19, decreased blood immunoglobulin G, nausea, and pyrexia.4-6

MG-ADL SCORES IN EXTENSION STUDIES

Consistent and clinically meaningful improvements in MG‑ADL scores across subsequent cycles of RYSTIGGO in the extension studies3

MG-ADL score across each subsequent 6-week treatment cycle3,6†

Consistent and clinically meaningful improvements in MG-ADL across each subsequent 6week treatment cycle.
Consistent and clinically meaningful improvements in MG-ADL across each subsequent 6week treatment cycle.

The most common adverse reactions being observed with repeated cyclic treatment were consistent with adverse reactions observed in the pivotal study1,6

Interim analysis as of July 8, 2022.5

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.3

Clinically meaningful was established as a ≥2.0-point improvement in MG-ADL total score.3

Treatment initiation on Day 1.3

MG-ADL OUTCOME MEASURE 

MG-ADL assesses the impact of gMG on daily functions of 8 symptoms on a scale of 0-3, with total scores ranging from 0-24. Higher scores are interpreted as greater impairments. An improvement of ≥2.0 points was established as clinically meaningful.1,2

Measures include7:

  • Voice/speech problems
  • Chewing
  • Swallowing
  • Breathing
  • Brushing teeth and/or combing hair
  • Rising from a chair
  • Diplopia
  • Eyelid droop

MG-ADL RESPONDER RATES IN EXTENSION STUDIES

Consistent MG-ADL responder rates across subsequent cycles of RYSTIGGO in the extension studies3

Proportion of adults with a ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43) without the use of rescue therapy3,6‖

MG-ADL responders had consistent and high response rates with subsequent cycles of RYSTIGGO.
MG-ADL responders had consistent and high response rates with subsequent cycles of RYSTIGGO.

Interim analysis as of July 8, 2022.3

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.3

QMG and MGC responders were established as adults with a ≥3.0-point improvement in QMG and MGC score from baseline at Week 6 (Day 43) without the use of rescue therapy.3,6

QMG responder rates from baseline at Week 6 (Day 43) of the extension studies were 68.5% (n=87/127) for cycle 1, 62.4% (n=78/125) for cycle 2, 64.9% (n=63/97) for cycle 3, 68.9% (n=51/74) for cycle 4, 58.8% (n=30/51) for cycle 5, and 65.6% (n=21/32) for cycle 6. MGC responder rates from baseline at Week 6 (Day 43) of the extension studies were 73.2% (n=93/127) for cycle 1, 74.8% (n=95/127) for cycle 2, 69.4% (n=68/98) for cycle 3, 73.3% (n=55/75) for cycle 4, 70.0% (n=35/50) for cycle 5, and 71.0% (n=22/31) for cycle 6.3

QMG OUTCOME MEASURE

QMG is a physician assessment scoring system that quantifies disease severity. Measures are assessed on a scale of 0-3, with total scores ranging from 0-39. An improvement of ≥3.0 points was established as clinically meaningful.1,2,8

Measures include8:

  • Ptosis
  • Facial muscle weakness
  • Dysarthria
  • Grip strength
  • Neck flexion endurance
  • Diplopia
  • Difficulty swallowing 4 oz of water
  • Percent predicted forced vital capacity
  • Arm and leg endurance

MGC OUTCOME MEASURE

MGC is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0-50. Higher scores are interpreted as greater impairments. An improvement of ≥3.0 points was established as clinically meaningful.2,9,10

Measures include9:

  • Ptosis
  • Eye closure
  • Chewing
  • Breathing
  • Shoulder abduction
  • Diplopia
  • Talking
  • Swallowing
  • Neck flexion or extension
  • Hip flexion

MG-ADL Response Rates in initial non-responders (post hoc analysis)11

MG-ADL response rates after subsequent cycles of RYSTIGGO in adults who were initial non-responders11††‡‡§§

Response observed in initial non-responders after additional RYSTIGGO cycles.
Response observed in initial non-responders after additional RYSTIGGO cycles.

Interim analysis as of July 8, 2022.11

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

This data was from a post hoc analysis not controlled for multiplicity and not powered; therefore, data should be interpreted with caution and conclusions cannot be drawn.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.11

MG-ADL responder was defined as adults with a ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43).11

26.0% of patients were initial non-responders in the MycarinG pivotal study.11

Cycle 1 only included adults from the RYSTIGGO treatment groups in MycarinG.11

MINIMAL SYMPTOM EXPRESSION (MSE) IN EXTENSION STUDIES

MSE rates through 6 subsequent cycles of RYSTIGGO3

Exploratory endpoint: Proportion of adults achieving MSE with each subsequent cycle of RYSTIGGO3¶¶

Adults who reached MSE achieved an MG-ADL total score of 0 to 1 at any visit for each 6week cycle and observation period without the use of rescue therapy during the extension studies.
Adults who reached MSE achieved an MG-ADL total score of 0 to 1 at any visit for each 6week cycle and observation period without the use of rescue therapy during the extension studies.

Interim analysis as of July 8, 2022.3

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

MSE was an exploratory endpoint and not controlled for multiplicity. Therefore, data should be interpreted with caution and conclusions cannot be drawn.

Data should be interpreted with caution given the decreasing number of patients receiving subsequent treatment cycles.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.3

Day 43.

MYASTHENIA GRAVIS SYMPTOMS PATIENT-REPORTED OUTCOME (MG SYMPTOMS PRO) IN EXTENSION STUDIES

Consistent improvements observed in gMG symptoms across 3 domains of MG Symptoms PRO12:

MG SYMPTOMS PRO OUTCOME MEASURE

MG Symptoms PRO is a novel outcome measure developed by UCB and used in the RYSTIGGO clinical trials. It complements established clinical outcome measures and is the first outcome measure to include a standalone assessment of physical fatigue.2,10,13

MG Symptoms PRO is a patient assessment evaluating the symptom severity of myasthenia gravis. Symptoms are measured over a 7-day period. A score ranging from 0-100 is calculated for each domain, with a higher score indicating greater symptom severity.10

MG Symptoms PRO evaluates2,10:

Muscle weakness fatigability A scale that measures muscle fatigability by assessing use-induced reduction in the ability of the following muscles to function:

  • Proximal
  • Ocular
  • Bulbar
  • Respiratory

Physical fatigue A scale that measures the symptoms/manifestations of physical fatigue, including:

  • Body and limb muscle weakness
  • Heaviness
  • Lack of energy and strength

Bulbar muscle weakness A scale that measures the symptoms/manifestations associated with bulbar muscle weakness, including:

  • Facial/mouth drooping
  • Speech and voice problems
  • Difficulties with chewing and swallowing
  • Liquid control in mouth
See a video that features Dr. Christyn Edmundson, an expert in gMG, discussing the RYSTIGGO extension studies that further evaluated the safety and efficacy of RYSTIGGO over time.
See a video that features Dr. Christyn Edmundson, an expert in gMG, discussing the RYSTIGGO extension studies that further evaluated the safety and efficacy of RYSTIGGO over time.

Hear the RYSTIGGO extension study results reviewed by an expert in gMG

WATCH VIDEO

TREATMENT-FREE INTERVALS

Majority of adults had treatment-free intervals between 4 and <8 weeks3

Time to subsequent symptom-driven treatment cycle from last subcutaneous infusion3,6§§§

Time to subsequent symptom-driven treatment cycle
Time to subsequent symptom-driven treatment cycle

Interim analysis as of July 8, 2022.3

  • The median time between treatment cycles was 8 weeks (56 days) for adults treated with RYSTIGGO who initiated 4 cycles1‖‖‖
  • The safety of initiating subsequent cycles sooner than 9 weeks (63 days) from the start of the previous treatment cycle has not been established1
  • The most common adverse reactions being observed with repeated cyclic treatment were consistent with adverse reactions observed in the pivotal study1,6

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.3

This is equivalent to 14 weeks (98 days) from the start of the previous treatment cycle.1

Learn more about RYSTIGGO flexible dosing with individualized treatment-free intervals

EXPLORE DOSING

Ab+=antibody positive; AChR=acetylcholine receptor; COVID-19=coronavirus disease 2019; gMG=generalized myasthenia gravis; IgG=immunoglobulin G; MG-ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGC=Myasthenia Gravis Composite; MGFA=Myasthenia Gravis Foundation of America; MuSK=muscle-specific tyrosine kinase; Nsub=number of participants with an MSE assessment; QMG=Quantitative Myasthenia Gravis.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7
  3. Bril V, Drużdż A, GrosskreutzJ, et al. Rozanolixizumab in generalized myasthenia gravis: Pooled analysis of the Phase 3 MycarinG study and two open-label extensions. J Neuromuscul Dis. 2025;12(2):218-230. doi:10.1177/22143602241305511
  4. A study to investigate the long-term safety, tolerability, and efficacy of rozanolixizumab in adult patients with generalized myasthenia gravis. ClinicalTrials.gov identifier: NCT04124965. Updated September 5, 2023. Accessed February 27, 2025. https://clinicaltrials.gov/study/NCT04124965
  5. A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis. ClinicalTrials.gov identifier: NCT04650854. Updated February 26, 2024. Accessed February 27, 2025. https://clinicaltrials.gov/study/NCT04650854
  6. Data on file. UCB, Inc., Smyrna, GA.
  7. MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/ADL.pdf
  8. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/QMG.pdf
  9. MG composite scale. Myasthenia Gravis Foundation of America. 2012. Accessed February 27, 2025. https://myasthenia.org/Portals/0/MG%20composite%20score.pdf
  10. Regnault A, Morel T, de la Loge C, et al. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms PRO. Neurol Ther. 2023;12(5):1573-1590. doi:10.1007/s40120-023-00464-x
  11. Pascuzzi RM, Grosskreutz J, Habib AA, et al. Response to rozanolixizumab across treatment cycles in patients with generalized myasthenia gravis: a post hoc analysis. Poster presented at: AAN 2024; April 13-18, 2024; Denver, CO. Poster P10-11-005.
  12. Habib AA, Kaminski HJ, Vissing J, et al. Patient-reported outcomes during repeated cycles of rozanolixizumab treatment in patients with generalized myasthenia gravis in the Phase 3 MycarinG and open-label extension studies. Poster presented at: MGFA Scientific Session 2023; November 1, 2023; Phoenix, AZ. Poster 21.
  13. Cleanthous S, Mork AC, Regnault A, et al. Development of the Myasthenia Gravis (MG) Symptoms PRO: a case study of a patient-centred outcome measure in rare disease. Orphanet J Rare Dis. 2021;16(1):1-14. doi:10.1186/s13023-021-02064-‍0

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MuSK+ SUBGROUP ANALYSIS

The efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG was established in an up to 18-week, multicenter, randomized, double-blind, placebo-controlled study. In the study, 200 patients were randomized 1:1:1 to receive weight-tiered doses of RYSTIGGO (n=133), either 7 mg/kg of RYSTIGGO (n=66) or 10 mg/kg of RYSTIGGO (n=67), or placebo (n=67). A subgroup analysis was performed evaluating adults with anti-MuSK Ab+ gMG.1,3

GO TO THE STUDY DESIGN 

MG-ADL IN ANTI-MuSK Ab+ ADULTS

The first treatment indicated for adults with anti-MuSK Ab+ gMG1

Subgroup analysis of the primary efficacy endpoint: Mean change from baseline to Week 6 (Day 43) in MG-ADL total score in adults with anti–MuSK Ab+ gMG3

After the 6-week treatment cycle, consistent improvements in MG-ADL scores in the RYSTIGGO treatment groups vs placebo were seen in adults with anti-MuSK Ab+ gMG.
After the 6-week treatment cycle, consistent improvements in MG-ADL scores in the RYSTIGGO treatment groups vs placebo were seen in adults with anti-MuSK Ab+ gMG.
After the 6-week treatment cycle, consistent improvements in MG-ADL scores in the RYSTIGGO treatment groups vs placebo were seen in adults with anti-MuSK Ab+ gMG.

All subgroup analyses were descriptive.3

MG-ADL OUTCOME MEASURE

MG-ADL assesses the impact of gMG on daily functions of 8 symptoms on a scale of 0-3, with total scores ranging from 0-24. Higher scores are interpreted as greater impairments. An improvement of ≥2.0 points was established as clinically meaningful.1,2

Measures include4:

  • Voice/speech problems
  • Chewing
  • Swallowing
  • Breathing
  • Brushing teeth and/or combing hair
  • Rising from a chair
  • Diplopia
  • Eyelid droop

MG-ADL RESPONDER RATE IN ANTI-MuSK Ab+ ADULTS

All adults with anti-MuSK Ab+ gMG who received RYSTIGGO and had data available at Week 6 (Day 43) were MG-ADL responders2

Subgroup analysis of MG-ADL responder rate: ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43) in adults with anti-MuSK Ab+ gMG2,3

100% of patients with anti-MuSK Ab+ gMG were clinical responders to RYSTIGGO (n=12).
100% of patients with anti-MuSK Ab+ gMG were clinical responders to RYSTIGGO (n=12).

All subgroup analyses were descriptive.3

Study limitations: MG-ADL responder rate was a prespecified secondary endpoint not controlled for multiplicity; therefore, data should be interpreted with caution and conclusions cannot be drawn.

Clinical responders were established as adults with a ≥2.0-point improvement in the total MG‑‍ADL score compared to baseline at Week 6 (Day 43). Clinical responder data were not collected for one patient in the RYSTIGGO 10 mg/kg group who discontinued treatment.2,5

QMG and MGC responders were established as adults with a ≥3.0-point improvement in QMG and MGC score from baseline at Week 6 (Day 43).3

QMG responder rates from baseline at Week 6 (Day 43) were 100% (n=5/5) in the RYSTIGGO 7 mg/kg group and 85.7% (n=6/7) in the RYSTIGGO 10 mg/kg group compared to 28.6% (n=2/7) in the placebo group. MGC responder rates from baseline at Week 6 (Day 43) were 100% (n=5/5) in the RYSTIGGO 7 mg/kg group and 100% (n=7/7) in the RYSTIGGO 10 mg/kg group compared to 0.0% (n=0/7) in the placebo group.3

QMG OUTCOME MEASURE

QMG is a physician assessment scoring system that quantifies disease severity. Measures are assessed on a scale of 0-3, with total scores ranging from 0-39. An improvement of ≥3.0 points was established as clinically meaningful.1,2,6

Measures include6:

  • Ptosis
  • Facial muscle weakness
  • Dysarthria
  • Grip strength
  • Neck flexion endurance
  • Diplopia
  • Difficulty swallowing 4 oz of water
  • Percent predicted forced vital capacity
  • Arm and leg endurance

MGC OUTCOME MEASURE

MGC is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0-50. Higher scores are interpreted as greater impairments. An improvement of ≥3.0 points was established as clinically meaningful.2,7,8 

Measures include7:

  • Ptosis
  • Eye closure
  • Chewing
  • Breathing
  • Shoulder abduction
  • Diplopia
  • Talking
  • Swallowing
  • Neck flexion or extension
  • Hip flexion

MINIMAL SYMPTOM EXPRESSION (MSE) IN ANTI-MuSK Ab+ ADULTS

MSE rates observed in adults with anti-MuSK Ab+ gMG during the pivotal study3

Subgroup analysis of MSE: Proportion of adults with anti-MuSK Ab+ gMG achieving MSE during treatment in the pivotal study3

Adults with anti-MuSK Ab+ gMG who reached MSE achieved an MG-ADL total score of 0 or 1 at any time up to and including Week 6 (Day 43) of the pivotal study.
Adults with anti-MuSK Ab+ gMG who reached MSE achieved an MG-ADL total score of 0 or 1 at any time up to and including Week 6 (Day 43) of the pivotal study.

All subgroup analyses were descriptive.3

MSE was an exploratory endpoint and not controlled for multiplicity. Therefore, data should be interpreted with caution and conclusions cannot be drawn.

MYASTHENIA GRAVIS SYMPTOMS PATIENT-REPORTED OUTCOME (MG SYMPTOMS PRO) IN ANTI-MuSK Ab+ ADULTS

MG Symptoms PRO evaluated gMG symptoms across 3 domains3:

MG SYMPTOMS PRO OUTCOME MEASURE

MG Symptoms PRO is a novel outcome measure developed by UCB and used in the RYSTIGGO clinical trials. It complements established clinical outcome measures and is the first outcome measure to include a standalone assessment of physical fatigue.2,8,9

MG Symptoms PRO is a patient assessment evaluating the symptom severity of myasthenia gravis. Symptoms are measured over a 7-day period. A score ranging from 0-100 is calculated for each domain, with a higher score indicating greater symptom severity.8

MG Symptoms PRO evaluates2,8:

Muscle weakness fatigability

A scale that measures muscle fatigability by assessing use-induced reduction in the ability of the following muscles to function:

  • Proximal
  • Ocular
  • Bulbar
  • Respiratory

Physical fatigue

A scale that measures the symptoms/manifestations of physical fatigue, including:

  • Body and limb muscle weakness
  • Heaviness
  • Lack of energy and strength

Bulbar muscle weakness

A scale that measures the symptoms/manifestations associated with bulbar muscle weakness, including:

  • Facial/mouth drooping
  • Speech and voice problems
  • Difficulties with chewing and swallowing
  • Liquid control in mouth

MG-ADL RESPONDER RATES IN ANTI-MuSK Ab+ ADULTS IN EXTENSION STUDIES

Consistent MG-ADL responder rates across subsequent cycles of RYSTIGGO in anti-MuSK Ab+ adults in the extension studies10

Subgroup analysis of MG-ADL responder rates in extension studies:

Proportion of adults with a ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43)10**

High response rates reported with subsequent cycles of RYSTIGGO in adults with anti-MuSK Ab+ gMG.
High response rates reported with subsequent cycles of RYSTIGGO in adults with anti-MuSK Ab+ gMG.

Interim analysis as of July 8, 2022.10

The RYSTIGGO extension studies were open label and not placebo controlled; therefore, the efficacy and clinical significance of the results should be interpreted with caution.

Data were pooled across MycarinG, the first 6 weeks of MG0004, and MG0007 and combined for the 7 mg/kg and 10 mg/kg RYSTIGGO treatment groups.10

QMG and MGC responders were established as adults with a ≥3.0-point improvement in QMG and MGC score from baseline at Week 6 (Day 43).10

QMG responder rates from baseline at Week 6 (Day 43) of the extension studies were 100% (n=12/12) for cycle 1, 83.3% (n=10/12) for cycle 2, 85.7% (n=6/7) for cycle 3, and 100% (n=6/6) for cycle 4. MGC responder rates from baseline at Week 6 (Day 43) of the extension studies were 100% (n=12/12) for cycle 1, 83.3% (n=10/12) for cycle 2, 71.4% (n=5/7) for cycle 3, and 83.3% (n=5/6) for cycle 4.10

Ab+=antibody positive; AChR=acetylcholine receptor; CI=confidence interval; gMG=generalized myasthenia gravis; IgG=immunoglobulin G; MG=myasthenia gravis; MG-ADL=Myasthenia Gravis Activities of Daily Living; MGC=Myasthenia Gravis Composite; MGFA=Myasthenia Gravis Foundation of America; MGSPRO=Myasthenia Gravis Symptoms Patient-Reported Outcome; MuSK=muscle-specific tyrosine kinase; QMG=Quantitative Myasthenia Gravis; SE=standard error.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7
  3. Habib AA, Sacconi S, Antonini G, et al. Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study. Ther Adv Neurol Disord. 2024;17:17562864241273036. doi:10.1177/17562864241273036
  4. MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/ADL.pdf
  5. Data on file. UCB, Inc., Smyrna, GA.
  6. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed February 27, 2025. https://myasthenia.org/Portals/0/QMG.pdf
  7. MG composite scale. Myasthenia Gravis Foundation of America. 2012. Accessed February 27, 2025. https://myasthenia.org/Portals/0/MG%20composite%20score.pdf
  8. Regnault A, Morel T, de la Loge C, et al. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms PRO. Neurol Ther. 2023;12(5):1573-1590. doi:10.1007/s40120-023-00464-x
  9. Cleanthous S, Mork AC, Regnault A, et al. Development of the Myasthenia Gravis (MG) Symptoms PRO: a case study of a patient-centred outcome measure in rare disease. Orphanet J Rare Dis. 2021;16(1):1-14. doi:10.1186/s13023-021-02064-0
  10. Habib AA, Sacconi S, Pascuzzi RM, et al. Repeated cycles of rozanolixizumab treatment in patients with muscle-specific kinase autoantibody-positive generalized myasthenia gravis. Poster presented at: AANEM 2023; November 1-4, 2023; Phoenix, AZ. Poster 203.

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