Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis

Topic:

Clinical Trials

Poster Number: 149

Author(s):

Tuan Vu, MD, University of South Florida, Andreas Meisel, MD, NeuroCure Clinical Research Center, Charité Universitätsmedizin Berlin, Renato Mantegazza, MD, Fondazione IRCCS Istituto Neurologico Carlo Besta, Djillali Annane, MD, Raymond Poincaré Hospital (AP-HP), Garches, France, Masahisa Katsuno, MD, Nagoya University Graduate School of Medicine, Nagoya, Japan, Rasha Aguzzi, Alexion, AstraZeneca Rare Disease, Boston, MA, USA, Nishi Rampal, Alexion, AstraZeneca Rare Disease, Boston, MA, USA, James Howard, MD, FAAN, The University of North Carolina at Chapel Hill

“Background: _x000D_
The 26-week, double-blind, randomized, placebo-controlled period (RCP) of the CHAMPION MG study (NCT03920293) demonstrated the efficacy and tolerability of the terminal complement C5 inhibitor ravulizumab in patients with anti-acetylcholine receptor antibody-positive (AChR Ab+) generalized myasthenia gravis (gMG). Patients who completed the RCP could receive ravulizumab in the ongoing open-label extension (OLE). _x000D_
Objective: _x000D_
To assess ravulizumab’s long-term efficacy and safety in adults with AChR Ab+ gMG._x000D_
Methods: _x000D_
In the OLE, patients receive intravenous ravulizumab (blind loading or bridging dose for those previously receiving placebo or ravulizumab, respectively, then 3000–3600 mg according to body weight every 8 weeks) for up to 4 years. Assessments include Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) total scores and safety evaluations. Interim analysis was conducted of data collected up to Week 60 from RCP baseline._x000D_
Results: _x000D_
Long-term efficacy and safety of ravulizumab were analyzed in 161 and 169 patients, respectively. Patients (n=83) who switched from placebo in the RCP to ravulizumab in the OLE showed rapid improvements in MG-ADL and QMG total scores, which were maintained through 34 weeks: least-squares mean (95% CI) changes from OLE baseline at Week 34 of the OLE (Week 60 from RCP baseline) were -1.7 (-2.7, -0.8; p=0.0007) and -3.1 (-4.2, -1.9; p<0.0001), respectively. Improvements achieved by ravulizumab-treated patients (n=78) in the RCP were sustained through 60 weeks: least-squares mean (95% CI) changes from RCP baseline at Week 60 were -4.0 (-4.8, -3.1; p<0.0001) and -4.1 (-5.4, -2.9; p<0.0001) for MG-ADL and QMG total scores, respectively. Ravulizumab was well tolerated; no meningococcal infections were reported. Four deaths occurred, all assessed by the investigators as unrelated to study treatment. _x000D_ Conclusion:_x000D_ Ravulizumab, administered every 8 weeks, demonstrated sustained improvements in MG symptoms and was well tolerated for up to 60 weeks in adults with AChR Ab+ gMG."