Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication VYVGART is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG). VYVGART is a neonatal Fc receptor blocker indicated for the treatment of adult patients with generalized myasthenia gravis (gMG). ( 1 ) FDA label
Also known as Vyvgart Hytrulo, Efgartigimod, efgartigimod alfa fcab, VYVGART
Efgartigimod Alfa · 17 trials · 27 indications
The percentage of patients who do not experience, at any time during the study, a 2 or more-point deterioration from their averaged baseline MG-ADL score that is sustained for a period of 4 or more consecutive weeks. (stable or improved patients)
The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (no symptoms) to 24 (most severe symptoms).
The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment.
Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study.
Number of enrolled participants per month
Proportion of randomized participants who receive both doses of assigned study intervention, attend all assigned study visits, and complete at least the high contrast visual acuity, low contrast visual acuity, and Pelli-Robson assessments at all visits
Difference in change in high-contrast visual acuity from baseline to 1 month between groups
Difference in change in low contrast visual acuity (LCVA) (# of letters seen at 2.5% illumination) from baseline to 1 month between groups
An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was any untoward medical occurrence that, at any dose: resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or any other medically important event. Treatment-emergent adverse events (TEAEs) were defined as AEs with onset on or after the first administration of study drug up to and including 60 days after the last study drug administration. Adverse events in the 'Infections and infestations' SOC were defined as AE of Special Interest (AESIs) because efgartigimod causes a transient reduction in total IgG levels.
A Composite of Relevant Endpoints for Sjögren's Syndrome (CRESS) responder is defined as improvements in at least 3 of the 5 items of CRESS (systemic disease activity, patient-reported symptoms, tear gland function, salivary gland function and serology. The score ranges from 0 to 9 (higher score = worse symptoms).
Composite Autonomic Symptom Score (COMPASS) 31 modified version (2-week recall) is a self-rated questionnaire to evaluate the severity and distribution of autonomic symptoms in various autonomic nerve disorders. It consists of 31 questions in 6 weighted domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal {GI}-mixed upper and diarrhea, bladder, and pupillomotor). A weighted total score of 0 (mild) to 100 (severe) was determined by adding a maximum raw score for each domain. Higher scores indicated a more severe degree of autonomic symptoms.
The Malmö POTS Symptom Score (MaPS) score is a dedicated POTS symptom scoring questionnaire. The score consists of 12 questions that assess symptom burden related (tachycardia, palpitations, dizziness, presyncope) and unrelated to orthostatic intolerance (GI symptoms, insomnia, concentration difficulties). Participants graded their symptoms for the past 7 days using a visual analog scale ranging from 0 (no symptoms) to 10 (worst possible). The total score was calculated by summing up the items/individual items and range was 0 to 120 points, with higher scores indicating more severe symptoms.
An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was any untoward medical occurrence that at any dose resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or any other medically important event. Treatment-emergent adverse events (TEAEs) were defined as AEs with onset on or after the first administration of study drug up to and including 60 days after the last study drug administration.
AE : adverse event; SAE : serious adverse event; AESI : adverse event of special interest
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Total Immunoglobulin G levels will be measured from blood samples
Total Immunoglobulin G (IgG) levels will be measured from blood samples
The proportion of patients who exhibit reduction in serum C7 antibody levels at week 26 as compared to week 1.
Occurrence of adverse events and effects.
The overall improvement of EB symptoms at week 26 as compared to week 1, measured by percentage change of a participant's EBDASI score (overall total score, total activity score, and total damage score). The EBDASI is scored in the range of 0 - 506, with a lower score corresponding to mild disease and higher score corresponding to more severe disease.
| Arm | Type | Description |
|---|---|---|
| treatment | EXPERIMENTAL | Efgartigimod infusion |
| Efgartigimod IV | EXPERIMENTAL | Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s) |
| Placebo IV | EXPERIMENTAL | Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s) |
| Placebo | PLACEBO_COMPARATOR | Patients receiving placebo during part A and receiving efgartigimod IV during part B |
| Efgartigimod IV - continuous regimen | EXPERIMENTAL | Participants receiving efgartigimod IV on a continuous regimen |
| Efgartigimod IV - cyclic regimen | EXPERIMENTAL | Participants receiving efgartigimod IV on a cyclic regimen |
| efgartigimod | EXPERIMENTAL | patients receiving efgartigimod |
| Efgartigmod alfa | ACTIVE_COMPARATOR | 10 patients will receive efgartigimod alfa All participants will receive standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks. |
| Efgartigimod IV + Empasiprubart IV | EXPERIMENTAL | Participants receive efgartigimod IV in part A, B and C and empasiprubart IV in part B |
| Efgartigimod IV (part A + C) | EXPERIMENTAL | Participants not eligible for part B, receiving efgartigimod IV in part A and C |
| Efgartigimod IV arm | EXPERIMENTAL | patients receiving infusions of Efgartigimod IV |
| Placebo arm | PLACEBO_COMPARATOR | patients receiving infusions of placebo IV |
| Efgartigimod or Efgartigimod PH20 SC | EXPERIMENTAL | Patients receiving Efgartigimod IV treatment or Efgartigimod PH 20 SC treatment |
| Mild renal impairment | EXPERIMENTAL | Patients with 60 \<= eGFR \<90 |
| Moderate renal impairment | EXPERIMENTAL | Patients with 30 \<= eGFR \<60 |
| Severe renal impairment | EXPERIMENTAL | Patients with eGFR \<30 |
| Normal renal function | EXPERIMENTAL | Patients with eGFR \>=90 |
| Name | Type | Description |
|---|---|---|
| efgartigimod administration | DRUG | efgartigimod infusion |
| Efgartigimod IV | BIOLOGICAL | Intravenous infusion of efgartigimod |
| Placebo IV | OTHER | Intravenous infusion of placebo |
| efgartigimod | BIOLOGICAL | Intravenous infusion of efgartigimod |
| Placebo | OTHER | Intravenous infusion of placebo |
| Efgartigimod Alfa | DRUG | 2,016 mg will be administered subcutaneously by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation. |
| Empasiprubart IV | BIOLOGICAL | Intravenous infusion of empasiprubart |
| Efgartigimod IV or Efgartigimod PH20 SC | BIOLOGICAL | Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC |
Inclusion Criteria: * 1\. Signed informed consent. 2. Age 18-80 years 3. Acetylcholine receptor antibody positive, myasthenia gravis patients, with stable disease for the past four or more months. Stable disease is defined as no change in dosage or interval in IVIG treatments and without any signif...
Efgartigimod alfa is being studied as a treatment for acute optic neuritis, a neurological condition involving inflammation of the optic nerve. It is an investigational therapy in Phase 2 clinical development and is not yet approved for this indication. The research focuses on patients 18 years and older.
Efgartigimod alfa targets FCGRT, the neonatal Fc receptor, and acts as an antagonist. By binding this receptor, it is designed to reduce levels of circulating IgG antibodies, including autoantibodies thought to contribute to inflammatory damage in conditions such as optic neuritis.
Efgartigimod alfa is being developed by argenx SE, a biotechnology company that trades under the ticker ARGX. The company is sponsoring the clinical research evaluating the drug in neurology indications, including acute optic neuritis.
Efgartigimod alfa is in Phase 2 clinical development for optic neuritis. The program is investigational and has not been approved by the FDA for this indication. The single Phase 2 trial is currently recruiting participants.
Efgartigimod alfa is being evaluated in the Phase 2 trial NCT06453694, titled Efgartigimod for the Treatment of Acute Optic Neuritis. The trial is recruiting, is placebo-controlled, randomized, and double-blind, and plans to enroll 20 participants in the United States.
Yes, efgartigimod alfa is described as a small molecule and is classified as an FCGRT antagonist. It is under investigation in neurology, specifically for acute optic neuritis, in a Phase 2 trial that is currently recruiting participants.