Myasthenia Gravis Treatments
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Generic Name
Efgartigimod
Brand Names
Vyvgart, VYVGART Hytrulo
FDA approval date: December 17, 2021
Classification: Endoglycosidase
Form: Injection
What is Vyvgart (Efgartigimod)?
VYVGART HYTRULO is indicated for the treatment of adult patients with: generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive chronic inflammatory demyelinating polyneuropathy VYVGART HYTRULO is a combination of efgartigimod alfa, a neonatal Fc receptor blocker, and hyaluronidase, an endoglycosidase, indicated for the treatment of adult patients with: generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive chronic inflammatory demyelinating polyneuropathy
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Efficacy and Safety of Nipocalimab vs Efgartigimod for Patients With Generalized Myasthenia Gravis in a Randomized, Open-label, Phase 3b, Interventional Trial Including Within Class Switching From Efgartigimod to Nipocalimab
Summary: The purpose of this study is to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis (a condition in which body's immune system mistakenly attacks and damages the connection between nerves and muscles causing muscle weakness).
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Brand Information
VYVGART (EFGARTIGIMOD ALFA)
1INDICATIONS AND USAGE
VYVGART is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG).
2DOSAGE FORMS AND STRENGTHS
Injection: 400 mg/20 mL (20 mg/mL) as a colorless to slightly yellow, clear to slightly opalescent solution, in a single-dose vial.
3CONTRAINDICATIONS
VYVGART is contraindicated in patients with serious hypersensitivity to efgartigimod alfa products or to any of the excipients of VYVGART
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described elsewhere in the labeling:
- Infections
- Hypersensitivity Reactions
- Infusion-Related Reactions
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
In clinical studies, the safety of VYVGART in adult patients with gMG has been evaluated in 364 patients who received at least one dose of VYVGART.
In a placebo-controlled study (Study 1) in patients with gMG, 84 patients received VYVGART 10 mg/kg
The minimum time to initiate a subsequent cycle, specified by study protocol, was 28 days from the last administration of the previous treatment cycle. On average, VYVGART-treated patients received 2 cycles in Study 1. The mean and median times to the second treatment cycle were 54 days and 50 days from the last administration of the first treatment cycle, respectively, for VYVGART-treated patients.
Adverse reactions reported in at least 5% of patients treated with VYVGART and more frequently than placebo are summarized in Table 1. The most common adverse reactions (reported in at least 10% of VYVGART-treated patients) were respiratory tract infection, headache, and urinary tract infection.
In the placebo-controlled study in patients with gMG who are anti-acetylcholine receptor (AChR) antibody negative (Study 2), 119 patients received one cycle of once-weekly administrations for 4 weeks of either VYVGART 10 mg/kg (n=58) or placebo (n=61). The overall safety profile in Study 2 was consistent with the known safety profile of VYVGART in patients with gMG, except for nausea, which occurred in 7% of anti-AChR antibody negative patients who received VYVGART compared to 5% of patients who received placebo.
The safety of initiating subsequent cycles between 7 and 28 days from the last administration of the previous treatment cycle was assessed in another study in 60 patients with gMG (78% AChR antibody positive and 22% anti-AChR antibody negative). Of these patients, 63% were exposed to treatment for over a year when cycles were initiated less than 4 weeks after the last administration. Safety in these patients was similar to that seen in Study 1.
4.2Postmarketing Experience
The following adverse reactions have been identified during postapproval use of VYVGART. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Immune System Disorders: Hypersensitivity reactions including anaphylaxis and hypotension, and infusion-related reactions
5DESCRIPTION
Efgartigimod alfa-fcab is a human immunoglobulin G1 (IgG1) -derived Fc fragment (fragment, crystallized) of the za allotype. The efgartigimod alfa-fcab Fc fragment is a homodimer consisting of two identical peptide chains each consisting of 227 amino acids linked together by two interchain disulfide bonds with affinity for FcRn. The molecular weight of efgartigimod alfa-fcab is approximately 54 kDa.
VYVGART (efgartigimod alfa-fcab) injection is a sterile, preservative free, clear to slightly opalescent, colorless to slightly yellow solution supplied in a single-dose vial for infusion after dilution.
Each 20 mL single-dose vial contains 400 mg of efgartigimod alfa-fcab at a concentration of 20 mg/mL. In addition, each mL of solution contains arginine hydrochloride (10.5 mg), histidine (1.4 mg), L- histidine hydrochloride monohydrate (2.2 mg), methionine (1.5 mg), polysorbate 80 (0.4 mg), sodium chloride (4.1 mg), sucrose (20.5 mg), and Water for Injection, USP, at a pH of 6.0.
6CLINICAL STUDIES
The efficacy of VYVGART for the treatment of adult patients with generalized myasthenia gravis (gMG) was established in two multicenter, randomized, double-blind, placebo-controlled trials, one in adults who are anti-AChR antibody positive (Study 1) and one in adults who are anti-AChR antibody negative (Study 2).
Study 1
The efficacy of VYVGART for the treatment of gMG in adults who are anti-AChR antibody positive was established in a 26-week, multicenter, randomized, double-blind, placebo-controlled trial (Study 1; NCT03669588).
Study 1 enrolled patients who met the following criteria at screening:
- Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IV
- MG-Activities of Daily Living (MG-ADL) total score of ≥ 5
- On stable dose of MG therapy prior to screening, that included acetylcholinesterase (AChE) inhibitors, steroids, or non-steroidal immunosuppressive therapies (NSISTs), either in combination or alone
- IgG levels of at least 6 g/L
A total of 167 patients were enrolled in Study 1 and were randomized to receive either VYVGART 10 mg/kg (1,200 mg for those weighing 120 kg or more) (n=84) or placebo (n=83). Baseline characteristics were similar between treatment groups. Patients had a median age of 46 years at screening (range: 19 to 81 years) and a median time since diagnosis of 7 years. Seventy-one percent were female, and 84% were White. Median MG-ADL total score was 9, and median Quantitative Myasthenia Gravis (QMG) total score was 16. The majority of patients (n=65 for VYVGART; n=64 for placebo) were positive for anti-AChR antibodies.
At baseline, over 80% of patients in each group received AChE inhibitors, over 70% in each treatment group received steroids, and approximately 60% in each treatment group received NSISTs, at stable doses.
Patients were treated with VYVGART at the recommended dosage regimen
The efficacy of VYVGART was measured using the Myasthenia Gravis-Specific Activities of Daily Living scale (MG-ADL) which assesses the impact of gMG on daily functions of 8 signs or symptoms that are typically affected in gMG. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. A total score ranges from 0 to 24, with the higher scores indicating more impairment. In this study, an MG-ADL responder was defined as a patient with a 2-point or greater reduction in the total MG-ADL score compared to the treatment cycle baseline for at least 4 consecutive weeks, with the first reduction occurring no later than 1 week after the last infusion of the cycle.
The primary efficacy endpoint was the comparison of the percentage of MG-ADL responders during the first treatment cycle between treatment groups in the anti-AChR antibody positive population. A statistically significant difference favoring VYVGART was observed in the MG-ADL responder rate during the first treatment cycle (67.7% in the VYVGART-treated group vs 29.7% in the placebo-treated group [p <0.0001]).
The efficacy of VYVGART was also measured using the Quantitative Myasthenia Gravis (QMG) total score which is a 13-item categorical grading system that assesses muscle weakness. Each item is assessed on a 4-point scale where a score of 0 represents no weakness and a score of 3 represents severe weakness. A total possible score ranges from 0 to 39, where higher scores indicate more severe impairment. In this study, a QMG responder was defined as a patient who had a 3-point or greater reduction in the total QMG score compared to the treatment cycle baseline for at least 4 consecutive weeks, with the first reduction occurring no later than 1 week after last infusion of the cycle.
The secondary endpoint was the comparison of the percentage of QMG responders during the first treatment cycle between both treatment groups in the anti-AChR antibody positive patients. A statistically significant difference favoring VYVGART was observed in the QMG responder rate during the first treatment cycle (63.1% in the VYVGART-treated group vs 14.1% in the placebo-treated group [p <0.0001]).
The results are presented in Table 2.
Figure 1 shows the mean change from baseline on the MG-ADL during cycle 1.
Figure 2 shows the distribution of response on the MG-ADL and QMG during cycle 1, four weeks after the first infusion with VYVGART.
Study 2
The efficacy of VYVGART for the treatment of adult patients with gMG who are anti-AChR antibody negative was established in an 8-week, randomized double-blind, placebo-controlled study (Study 2 Part A; NCT06298552). Patients were treated with VYVGART with the recommended dosage regimen
Study 2 enrolled patients with gMG who met the following criteria at screening:
- MGFA Clinical Classification Class II to IV
- MG-ADL total score of at least 5
- On stable doses of MG therapy prior to screening, that included AChE inhibitors, steroids or NSISTs, either in combination or alone
- IgG levels of at least 4 g/L
In Study 2 Part A, a total of 119 patients were randomized 1:1 to receive once-weekly infusions for 4 weeks of either VYVGART 10 mg/kg (1,200 mg for those weighing 120 kg or more) (n=58) or placebo (n=61). Subsequent treatment cycles were administered in the open-label Part B of the study per the recommended dosage regimen for two cycles followed by variable inter-treatment periods no shorter than 7 days for additional cycles for up to two years.
Baseline characteristics were similar between treatment groups. The median age was 50 years at screening (range: 21 to 80 years; median 49 years for patients treated with VYVGART and 52 years for placebo) and the median time since diagnosis was 4 years. Seventy six percent of patients were female, and 80% were White, 14% were Asian, and 3% were Black or African American. At baseline, the median MG-ADL total score was 9, and median QMG total score was 14.
At baseline, over 75% of patients in each treatment group received AChE inhibitors and over 60% in each treatment group received steroids at stable doses. There were more patients receiving NSISTs in the VYVGART arm (53%) than those in the placebo arm (36%).
Sixty-one percent of patients were triple seronegative (i.e., anti-AChR, anti-MuSK, and anti-low-density lipoprotein receptor-related protein 4 [LRP4] antibodies negative). Thirty-four percent of patients were positive for anti-MuSK antibodies, and 5% of patients were positive for anti-LRP4 antibodies.
The primary efficacy endpoint was the comparison of the mean change from baseline at Week 4 between treatment groups in the MG-ADL total score. A statistically significant difference favoring VYVGART was observed (LS mean difference [95% CI] = - 1.46 [-2.50 to -0.41]; p-value = 0.0068).
Figure 3 shows the mean change from baseline to Week 8 in MG-ADL total score in Study 2 Part A.
7HOW SUPPLIED/STORAGE AND HANDLING
VYVGART (efgartigimod alfa-fcab) injection is a preservative free, sterile, colorless to slightly yellow, clear to slightly opalescent solution supplied as 400 mg/20 mL (20 mg/mL) in one single-dose vial per carton (NDC 73475-3041-5).
8PRINCIPAL DISPLAY PANEL - 400 mg/20 mL Vial Carton
NDC 73475-3041-5
VYVGART
400 mg/20 mL
For intravenous use only
Rx only
