Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Efgartigimod IV
Efgartigimod · 16 trials · 26 indications
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.
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.
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 |
|---|---|---|
| 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 |
| 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 |
| 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 |
| Efgartigimod-1 | EXPERIMENTAL | Weekly efgartigimod infusions and the pneumovax 23 vaccine on day 22 |
| Efgartigimod-2 | EXPERIMENTAL | Weekly efgartigimod infusions and the pneumovax 23 vaccine on day 36 |
| efgartigimod PH20 SC | EXPERIMENTAL | subcutaneous injections of efgartigimod PH20 SC |
| Name | Type | Description |
|---|---|---|
| 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 |
| Empasiprubart IV | BIOLOGICAL | Intravenous infusion of empasiprubart |
| PNEUMOVAX 23 | BIOLOGICAL | PNEUMOVAX 23 vaccine |
| efgartigimod PH20 SC | BIOLOGICAL | subcutaneous injections of efgartigimod PH20 SC |
Inclusion Criteria: * Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF). * Has documented baseline mean platelet count of \<30 x 10\^9/L before randomization * Has a documented duration of primary immune thrombocytopenia...
Efgartigimod is an investigational monoclonal antibody being studied for several conditions, including Primary Sjögren's Syndrome, Primary Immune Thrombocytopenia (ITP), Postural Orthostatic Tachycardia Syndrome, and AChR-Ab Seropositive Generalized Myasthenia Gravis. It is also being evaluated in healthy volunteers in early-phase trials. As of the current data, it remains in clinical development and is not yet approved.
Efgartigimod is being developed by argenx SE, a biopharmaceutical company traded on the stock exchange under the ticker ARGX. The company is conducting clinical trials to evaluate the drug's safety and efficacy across multiple indications, including immune thrombocytopenia and generalized myasthenia gravis.
Efgartigimod is currently in Phase 2 clinical development. The most advanced trials include a Phase 2 study in adolescents with chronic immune thrombocytopenia and a Phase 2 study evaluating it as an add-on therapy in generalized myasthenia gravis. Earlier Phase 1 trials in healthy volunteers have been completed.
Efgartigimod is being studied in several clinical trials. NCT07194850 is a Phase 2 trial in adolescents with chronic ITP, currently recruiting. NCT07284420 is a Phase 2 trial in adults with AChR-Ab seropositive generalized myasthenia gravis. Completed Phase 1 trials include NCT04564066 and NCT05163834, both in healthy volunteers.
Yes, Efgartigimod is also known as Efgartigimod IV, referring to the intravenous formulation being studied. Clinical trials such as NCT04564066 compare the intravenous infusion with a subcutaneous injection. The drug is being evaluated in both healthy volunteers and patient populations across multiple indications.
Efgartigimod is a monoclonal antibody designed to target and block the neonatal Fc receptor (FcRn). By inhibiting FcRn, it reduces circulating IgG antibody levels, which may help in autoimmune conditions where pathogenic IgG antibodies drive disease. This mechanism is being studied in conditions like ITP and myasthenia gravis.