Long-term safety, tolerability, and efficacy of subcutaneous efgartigimod PH20 in generalized myasthenia gravis: final results from a phase 3 open-label extension study (ADAPT-SC+) - Report - MDSpire
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Long-term assessment of safety, tolerability, and effectiveness of subcutaneous efgartigimod PH20 in adults with generalized myasthenia gravis: final findings from the phase 3 open-label extension study (ADAPT-SC+)
Clinical Report: Long-term assessment of efgartigimod PH20 in gMG
Overview
The ADAPT-SC+ study evaluated the long-term safety, tolerability, and efficacy of subcutaneous efgartigimod PH20 in adults with generalized myasthenia gravis (gMG). Results indicated that 95.1% of participants with AChR-Ab+ gMG achieved clinically meaningful improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores.
Background
Generalized myasthenia gravis (gMG) is an autoimmune disorder characterized by fluctuating muscle weakness, often leading to severe complications. Current treatments primarily provide symptomatic relief rather than addressing the underlying mechanisms of the disease. Efgartigimod, a neonatal Fc receptor antagonist, represents a novel therapeutic approach aimed at reducing pathogenic IgG autoantibodies in gMG.
Data Highlights
Parameter
Result
Participants enrolled
184
Participants receiving treatment
180
Mean treatment duration
2.6 years
TEAEs reported
93.9%
Injection site reactions
46.1%
COVID-19 cases
29.4%
Headache cases
26.7%
CMI in AChR-Ab+ participants
95.1%
CMI in AChR-Ab− participants
94.7%
MSE in AChR-Ab+ participants
59.2%
MSE in AChR-Ab− participants
34.2%
Key Findings
93.9% of participants experienced treatment-emergent adverse events (TEAEs).
Injection site reactions were the most common TEAEs, reported by 46.1% of participants.
95.1% of AChR-Ab+ participants achieved clinically meaningful improvement (CMI) in MG-ADL scores.
59.2% of AChR-Ab+ participants attained minimal symptom expression (MSE).
More frequent dosing did not introduce new safety signals.
83.3% of participants with AChR-Ab+ gMG maintained MSE across consecutive assessments for ≥8 weeks.
Clinical Implications
The findings support the long-term safety and efficacy of efgartigimod PH20 in treating gMG, indicating its potential as a viable treatment option for both AChR-Ab+ and AChR-Ab− patients. The ability to individualize treatment cycles may enhance patient management.
Conclusion
Efgartigimod PH20 demonstrates sustained efficacy and a favorable safety profile in adults with gMG, reinforcing its role in the long-term management of this condition.
by Andreas Meisel, Carlo Antozzi, Ratna Bhavaraju-Sanka, Jan L. De Bleecker, Wan-Yi Huang, Rosa Hermina Jimenez, Fien M. Verhamme, Ming Jiang, Li Liu, Denis Korobko, Anna Kostera-Pruszczyk, Kimiaki Utsugisawa, Jan J. G. M. Verschuuren, Tuan Vu, Heinz Wiendl, James F. Howard
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