https://orcid.org/0000-0003-3347-9751
![Sample our Medicine, Dentistry, Nursing & Allied Health journals, sign in here to start your FREE access for 14 days]({"type":"image" , "src" : "/sda/5944/TOC-Subject-Sample-2021-Medicine-Dentistry-Nursing-Allied-Health.jpg"})
ABSTRACT
Introduction
Myasthenia gravis (MG) is an auto-immune disease characterized by fluctuating symptoms of muscle weakness and fatigue. Corticosteroids and corticosteroid-sparing broad-spectrum immunosuppression play a great role in the treatment of myasthenia gravis. However, debilitating side effects and long time to treatment effect highlight the need for development of novel target-specific medications. Rozanolixizumab is a highly specific neonatal Fc receptor (FcRn) inhibitor that acts on immunoglobulin G (IgG) homeostasis. Results from the MycarinG Phase III randomized controlled trial demonstrated significant efficacy of rozanolixizumab in generalized MG in terms of primary outcome and all secondary endpoints, tolerability, and safety compared to placebo.
Areas covered
We included different trials on myasthenia gravis and rozanolixizumab which include Phase II (NCT03052751) and Phase III MycarinG (NCT03971422) studies.
Expert opinion
Clinical trials have demonstrated that rozanolixizumab has strong efficacy with a 78% reduction in pathogenic IgG like plasma exchange (PLEX) and has therapeutic benefits comparable with PLEX and IVIG. It has less treatment adverse events and is easily accessible through subcutaneous infusion. The safety and effectiveness of rozanolixizumab need to be assessed further in the real-world context in post-marketing studies. If current trial information holds true, rozanolixizumab may become a medication of choice for MG in succeeding years.
Article highlights
Rozanolixizumab is a novel FcR inhibitor that reduces levels of IgG.
Rozanolixizumab reduces levels of pathogenic AChR and MuSK antibodies in MG and is the only FcR inhibitor approved by the FDA for the treatment of both MuSK and AChR antibody positive MG.
Rozanolixizumab is administered subcutaneously avoiding the need for regular intravenous infusions.
In the pivotal phase III MG trial, rozanolixizumab improved multiple outcomes including MG-ADL, QMG, MGC and MG-PRO.
Rozanolixizumab was well-tolerated with headache being the most frequent adverse event.
List of Abbreviations
| AChRAb | = | acetylcholine receptor (AChR) antibody |
| CIDP | = | Chronic Inflammatory Demyelinating Polyneuropathy |
| EMA | = | European Medicine Agency |
| FcRn | = | Neonatal Fc Receptors |
| FDA | = | Food and Drug Administration |
| IgG | = | Immunoglobulin G |
| iRODS | = | inflammatory Rasch-built overall disability scale score |
| ITP | = | Primary Immune Thrombocytopenia |
| ITP-PAQ | = | ITP Patient Assessment Questionnaire |
| IV | = | Intravenous |
| IVIg | = | Intravenous Immunoglobulin |
| LG-1 | = | Leucine-Rich Glioma Inactivated 1 |
| LRP 4 | = | lipoprotein receptor-related 4 |
| LS | = | Least Squares |
| MG | = | Myasthenia Gravis |
| MG- ADL | = | Myasthenia Gravis Activities of Daily Living Profile |
| MGC | = | MG Composite |
| MHC | = | major histocompatibility complex |
| MHLW | = | Ministry of Health, Labour and Welfare |
| MOG-AD | = | Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease |
| MuSK | = | muscle specific tyrosine kinase antibody |
| PLEX | = | plasma exchange |
| QMG | = | Quantitative Myasthenia Gravis |
| SC | = | Subcutaneously |
| TEAE | = | treatment-emergent adverse events |
| TGA | = | Therapeutic Goods Administration |
| UCL | = | Upper confidence limit |
Declaration of interest
V Bril declares consultancy for Grifols, CSL, UCB, Argenx, Takeda, Alnylam Octapharma, Pfizer, Powell Mansfield Inc, Akcea, Ionis, Immunovant, Sanofi, Momenta (J&J), Roche, Janssen, AZ-Alexion, NovoNordisk and Japan Tobacco; and research support from AZ-Alexion, Grifols, CSL, UCB, Argenx, Takeda, Octapharma, Akcea, Momenta (J&J) and Immunovant, Ionis. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Reviewer disclosures
A reviewer on this manuscript has disclosed that they have been involved in various advisory board capacities for several drugs which work on similar mechanisms. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.