Argenx — Vyvgart EMA EPAR public assessment report (EMEA/H/C/005849, 2022)
Argenx · 2022 · EMA EPAR public assessment report · company profile →
Report summary
The EMA's assessment of Argenx's Vyvgart (efgartigimod alfa) for generalized Myasthenia Gravis (gMG) resulted in a positive CHMP opinion on June 23, 2022. Designated an orphan medicinal product, Vyvgart is indicated as an add-on therapy for adult anti-acetylcholine receptor (AChR) antibody positive gMG patients. Manufacturing quality, stability, and non-clinical pharmacology were thoroughly reviewed, confirming an 18-month shelf-life and low risks for contaminants. Non-clinical studies demonstrated efgartigimod's FcRn antagonist mechanism, reducing IgG levels and improving gMG symptoms in animal models with acceptable safety. Pharmacokinetic, pharmacodynamic, and toxicology studies established NOAELs without significant human-translatable safety signals. The pivotal Phase 3 ARGX-113-1704 trial showed significant improvements in MG-ADL and QMG responder rates in AChR-Ab seropositive patients. While generally well-tolerated, common adverse events included headache and infections. Long-term safety data is limited, and a Risk Management Plan is in place to further characterize risks, particularly regarding long-term use and special populations. The regulatory indication was narrowed to AChR-Ab seropositive patients due to insufficient data in seronegative individuals.
Key findings — cited to the page
| TEAE of CTCAE severity Grade ≥3 in females | 16.2 % | · p.159 |
| Lymphocyte count decreased CTCAE Grade 3 (Control) | 9.6 % | ARGX-113-1704 · p.161 |
| TEAE of CTCAE severity Grade ≥3 in males | 28.9 % | · p.159 |
| Lymphocyte count decreased CTCAE Grade 3 | 9.5 % | ARGX-113-1704 · p.161 |
| Severe (grade ≥3) TEAEs in males | 28.9 % | · p.149 |
| TEAEs of CTCAE severity Grade ≥3 in patients ≥65 years | 28.0 % | PB2 · p.133 |
| TEAEs of CTCAE severity grade ≥3 (placebo) | 9.6 % | ARGX-113-1704 · p.144 |
| TEAEs of CTCAE severity Grade ≥3 in patients <65 years | 18.2 % | PB2 · p.133 |
| Severe (grade ≥3) TEAEs in patients ≥65 years | 28.0 % | · p.149 |
| Severe (grade ≥3) TEAEs in patients <65 years | 18.2 % | · p.149 |
| Severe (grade ≥3) TEAEs in females | 16.2 % | · p.149 |
| TEAEs of CTCAE severity grade ≥3 (efgartigimod) | 10.7 % | ARGX-113-1704 · p.144 |
Source: Argenx ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Immunology library →