DOI: 10.1177/17562864261458532 ISSN: 1756-2864

Long-term use of rozanolixizumab in generalised myasthenia gravis: final pooled analysis of the phase III MycarinG study and two open-label extensions

Vera Bril, Artur Drużdż, Julian Grosskreutz, Ali A. Habib, Renato Mantegazza, Sabrina Sacconi, Kimiaki Utsugisawa, Tuan Vu, Marion Boehnlein, Fiona Grimson, Niamh Houston, Virginie Kerbusch, Irene Pulido-Valdeolivas, Thaïs Tarancón, John Vissing
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Background:

Myasthenia gravis (MG) is a rare autoimmune disease characterised by fluctuating and fatigable muscle weakness. In the randomised, double-blind phase III MycarinG study, one 6-week rozanolixizumab cycle significantly improved MG-specific outcomes versus placebo and was generally well tolerated in patients with generalised MG (gMG).

Objectives:

To assess the efficacy and safety of cyclic rozanolixizumab treatment.

Design:

A pooled analysis of the MycarinG, MG0004 and MG0007 studies.

Methods:

Following MycarinG, eligible patients could enrol in the open-label extension studies MG0004 or MG0007 to receive rozanolixizumab 7 or 10 mg/kg. In MG0004, patients received chronic weekly treatment for ⩽52 weeks. In MG0007, after an initial 6-week treatment cycle, subsequent cycles were based on symptom worsening (investigator’s discretion). Final efficacy data were pooled across MycarinG, MG0004 (first 6 weeks) and MG0007 for patients receiving ⩾2 symptom-driven cycles. Efficacy endpoints included change from baseline (CFB) in MG Activities of Daily Living (MG-ADL), MG Composite (MGC) and Quantitative MG (QMG) scores. Safety outcomes were assessed in patients who received ⩾1 cycle with a ⩽8-week follow-up period across MycarinG and MG0007.

Results:

Overall, 188 patients received ⩾1 cycle and 129 received ⩾2 symptom-driven cycles. Across Cycles 1–13, mean (standard deviation) CFB to Day 43 in MG-ADL score ranged from −3.2 (3.3 ( n  = 113; Cycle 3)) to −6.0 (3.9 ( n  = 24; Cycle 12)). Consistent improvements in MGC and QMG scores were also observed across repeated cycles. Treatment-emergent adverse events (TEAEs) were experienced by 175/188 (93.1%) patients; most mild or moderate. Incidence remained stable with repeated cyclic treatment among patients who remained in the study at each cycle. The most common TEAE was headache ( n  = 94/188 (50.0%)).

Conclusion:

Repeated rozanolixizumab treatment cycles demonstrated consistent, clinically meaningful improvements in MG-specific outcomes as early as 1 week after the first infusion. Rozanolixizumab was generally well tolerated with an acceptable safety profile, supporting its long-term use as a treatment option for adults with gMG.

Trial registration:

ClinicalTrials.gov: NCT03971422; NCT04124965; NCT04650854.

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