Uplizna (inebilizumab-cdon) / Mitsubishi Tanabe, Jiangsu Hansoh Pharma, Amgen - LARVOL DELTA

On 11 December 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Uplizna. The marketing authorisation holder for this medicinal product is Amgen Europe B.V. (European Medicines Agency) - "The CHMP adopted a new indication as follows: Neuromyelitis optica spectrum disorders (NMOSD)...Uplizna is indicated for the treatment of adult patients with active IgG4 RD....Uplizna is indicated as an add-on to standard therapy for the treatment of gMG in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive."

CHMP • Immunology • Myasthenia Gravis • Neuromyelitis Optica Spectrum Disorder

Amgen…announced that the U.S. Food and Drug Administration (FDA) has approved UPLIZNA (inebilizumab-cdon) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. (Amgen Press Release) - "The approval of UPLIZNA for gMG is supported by data from the Myasthenia Gravis Inebilizumab Trial (MINT), the largest Phase 3 biologic study to include both AChR+ and MuSK+ patients, and the first to successfully incorporate a steroid taper into its protocol."

FDA approval • Myasthenia Gravis

Inebilizumab: Pediatric Case Series in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Spectrum Disorder. (PubMed, Pediatr Neurol) - "This study adds to the literature because it shows that in our cohort inebilizumab was a safe medication for pediatric patients with NMOSD and prevented further relapses."

Journal • CNS Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Pediatrics • Rare Diseases

Consensus recommendations for the diagnosis and management of neuromyelitis optica spectrum disorder: A Saudi expert panel review. (PubMed, Mult Scler Relat Disord) - "This expert consensus provides evidence-based guidance for the diagnosis and management of NMOSD in Saudi Arabia, striking a balance between clinical evidence and expert opinion where data gaps exist. It emphasises individualised care, antibody testing, and long-term immunosuppressive strategies while highlighting the need for further research on treatment duration and failure criteria."

Journal • Review • CNS Disorders • Immunology • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

A recalcitrant case of pemphigus treated with inebilizumab. (PubMed, JAAD Case Rep) - No abstract available

Journal • Dental Disorders • Dermatology • Immunology • Oncology • Oral Cancer • Pemphigus Vulgaris • Stomatitis

What is in the Neuromuscular Junction Literature? (PubMed, J Clin Neuromuscul Dis) - "The role of thymectomy in older patients with MG is considered. The medical treatment section addresses corticosteroid regimens, intravenous immunoglobulin as maintenance therapy, a phase 3 study of the recently approved neonatal Fc receptor (FcRN) blocker nipocalimab, use of complement inhibitors and FcRN blockers in general, regimens for efgartigimod, and positive studies on the interleukin-16 receptor monoclonal antibody (Ab) satralizumab and the CD19 B-cell-depleting monoclonal Ab inebilizumab."

Journal • Review • CNS Disorders • Myasthenia Gravis • Ophthalmology • Thyroid Eye Disease • IL16

IgG4-Related disease: From diagnosis to remission. (PubMed, Best Pract Res Clin Rheumatol) - "Treatment strategies have evolved significantly with the introduction of B cell depletion therapy, particularly inebilizumab...Critical knowledge gaps persist regarding optimal biomarkers for disease activity, standardized remission criteria, and the precise etiopathogenesis of IgG4-RD. Despite therapeutic advances, challenges remain in balancing effective disease control with minimizing the harms of long-term immunosuppression, emphasizing the need for continued research into targeted therapies."

Journal • Review • Fibrosis • Immunology • Inflammation • Pancreatitis

Risk of hepatitis B and tuberculosis reactivation in patients with neuromyelitis optica spectrum disorder undergoing B cell depletion therapy. (PubMed, Mult Scler Relat Disord) - "Patients with NMOSD who were HBsAg-negative and anti-HBc-positive or had LTBI had no observed instances of HBVr/TBr while receiving B cell depletion therapy during the follow-up period, with or without prophylactic anti-HBV or anti-TB treatment."

Journal • CNS Disorders • Hepatitis B • Infectious Disease • Inflammation • Neuromyelitis Optica Spectrum Disorder • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Tuberculosis

Inebilizuma treatment for acute serum AQP4 - IgG neuromyelitis optica: Case report. (PubMed, Medicine (Baltimore)) - "This case demonstrates that combination therapy with IVMP and inebilizumab during the acute phase significantly promoted visual recovery in this AQP4-IgG seropositive NMOSD patient. This finding enhances our understanding of NMOSD management. The case offers valuable clinical insights for therapeutic strategies in NMOSD. Future studies with larger cohorts are warranted to further evaluate the efficacy of the intravenous methylprednisolone pulse-inebilizumab combination in reducing relapse rates and improving long-term neurological outcomes in NMOSD."

Journal • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Evaluating inebilizumab in the treatment of generalized myasthenia gravis. (PubMed, Immunotherapy) - "As the treatment options for MG expands, choosing the right treatment for the patient will rely on thoroughly evaluating the efficacy, time to achieve minimal symptomatic state, comparative effectiveness, treatment burden, side effects, and cost. Ultimately, data from clinical practice and ongoing phase III trials will be essential to define the best role of inebilizumab in treatment of MG."

Journal • Review • CNS Disorders • Myasthenia Gravis

Anti-CD19 monoclonal antibody UPLIZNA for I.V. Infusion 100mg Approval for additional indication of suppression of relapse of immunoglobulin G4-related disease in Japan (Firstwordpharma Press Release) - "A Phase 3 multi-regional clinical study (MITIGATE study) was conducted in collaboration with Amgen Inc., the company that developed UPLIZNA, to evaluate the efficacy and safety of UPLIZNA in patients with IgG4-RD. The approval is based on the data of MITIGATE study."

Japan approval • Immunology

IgG4-Mediated Interstitial Nephritis Treated with Inebilizumab (KIDNEY WEEK 2025) - "Prednisone was initiated and tapered, and the patient transitioned to inebilizumab infusions. Steroid sparing regimens are desirable to mitigate these consequences. Promising new drugs, such as inebilizumab which targets CD19 cells, have been shown to reduce flares in IgG4-RD and increase the chance of flare-free remission at one year."

Fibrosis • Immunology • Nephrology • Renal Disease

Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy (clinicaltrials.gov) - P=N/A | N=60 | Recruiting | Sponsor: Amgen | Trial completion date: Jul 2033 ➔ Oct 2032

Trial completion date • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG) (clinicaltrials.gov) - P2 | N=15 | Recruiting | Sponsor: Amgen | Initiation date: Nov 2025 ➔ Apr 2026

Trial initiation date • CNS Disorders • Myasthenia Gravis

Efficacy and Safety of Modern Biologics Compared to Conventional Therapies in AQP-4 Positive Neuromyelitis Optica Spectrum Disorder: A Fully AI-Automated Targeted Literature Review (ISPOR-EU 2025) - "Eculizumab, satralizumab, and inebilizumab showed significantly reduced annualized relapse rates (84-98%) compared to rituximab (74-100%) and traditional immunosuppressants (33-96%). Modern biologics show significantly better safety profile and superior efficacy in preventing relapses in adult AQP4-positive NMOSD patients compared to conventional therapies. The AI-based automated literature review by our in-house toolkit demonstrates the power of AI in performing complete reviews within 2-3 hours compared to a few weeks by manual processes. The AI pathways and efficacy comparison with manual processes will be presented."

Clinical • Review • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Use of Dupilumab as a Steroid-Sparing Agent in IgG4-Related Disease (ACAAI 2025) - "Despite hydroxychloroquine twice daily, he was unable to taper prednisone less than 5mg daily, and chronic steroid use had already resulted in steroid-induced osteopenia. He had failed other immunosuppressive agents: rituximab resulted in a severe transfusion reaction, mycophenolate mofetil caused recurrent infections, and azathioprine induced neutropenia. He was reluctant to start the anti-CD19 monoclonal antibody inebilizumab, as it had only just been approved for IgG4-RD treatment...This case highlights the potential utility of DUP as a steroid-sparing agent in IgG4-RD, particularly in patients with type 2 inflammatory comorbidities and contraindications to standard immunosuppressants. Further studies are needed to define its role in broader IgG4-RD management."

Asthma • Hematological Disorders • Infectious Disease • Inflammation • Neutropenia • Otorhinolaryngology • Respiratory Diseases • Sinusitis • IL13 • IL4

Rare Disease (Amgen Press Release) - "UPLIZNA (inebilizumab-cdon) sales increased 46% year-over-year to $155 million in the third quarter, primarily driven by volume growth. Year-over-year sales were impacted by the timing of shipments to our ex- U.S. partner, as shipments similar to those that occurred in the third quarter of 2024 occurred in the second quarter of 2025...TAVNEOS (avacopan) sales increased 34% year-over-year to $107 million in the third quarter, driven by 66% volume growth, partially offset by 16% lower inventory levels and 10% lower net selling price."

Sales • ANCA Vasculitis • Myasthenia Gravis

Optimizing therapeutic strategies: a prospective exploratory pilot study of different B-cell clearance therapies versus glucocorticoids in the induction and maintenance of refractory or relapsed autoimmune pancreatitis (ChiCTR) - P=N/A | N=48 | Not yet recruiting | Sponsor: Peking Union Medical College Hospital; Peking Union Medical College Hospital

New trial • Immunology • Pancreatitis

A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG) (clinicaltrials.gov) - P2 | N=15 | Recruiting | Sponsor: Amgen | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Inebilizumab in Pediatric Participants With IgG4-RD (clinicaltrials.gov) - P2 | N=15 | Not yet recruiting | Sponsor: Amgen

New P2 trial • Inflammation • Pediatrics

Inebilizumab Efficacy and Safety in Patients with Common, Urgent, and Fibrotic Organ Manifestations of IgG4-RD: Subgroup Analyses from the MITIGATE Trial (ACR Convergence 2025) - P3 | "The MITIGATE trial demonstrates consistent safety and efficacy of INEB across all IgG4-RD subgroups defined on the basis of organ involvement at study baseline, including the most common organs, urgent organs, and organs characteristic of the fibrotic disease phenotype."

Clinical • Fibrosis • Inflammation

Incidence of Coronavirus Disease 2019 in Participants with IgG4-Related Disease Treated with Inebilizumab in the MITIGATE Study (ACR Convergence 2025) - P3 | "Treatment with INEB was not associated with a higher incidence or severity of COVID-19 compared with placebo. Despite B-cell depletion, infections were mostly mild, and serious infections were infrequent. These findings support the safety of INEB in the context of the COVID-19 pandemic."

Infectious Disease • Inflammation • Novel Coronavirus Disease • Respiratory Diseases

Assessment of Baseline igg4-rd Disease Characteristics and Impact upon Safety and Efficacy of Inebilizumab: Results from the MITIGATE Study (ACR Convergence 2025) - P3 | "The MITIGATE trial demonstrates the consistent benefit and safety of inebilizumab across all baseline disease characteristic subgroups analyzed. Inebilizumab represents a viable treatment option for all patients, regardless of their IgG4-RD baseline disease characteristics."

Clinical • Inflammation • CD20

Clinical and Biomarker Characteristics of Inebilizumab-Treated Participants Who Experienced an IgG4-RD Flare During the Phase 3 MITIGATE Trial (ACR Convergence 2025) - P3 | "In this small cohort, baseline clinical and serologic characteristics did not reliably differentiate INEB-treated participants who experienced a flare (n=7) from those who did not (n=61). In INEB-treated participants, no clear patterns of biomarker changes are associated with occurrence of flare. Some participants experienced full apparent pharmacodynamic effect of INEB yet experience a flare."

Biomarker • Clinical • P3 data • CNS Disorders • Inflammation • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Neuromyelitis optica spectrum disorder (NMOSD): Recent advances and insights from Taiwan. (PubMed, J Formos Med Assoc) - "Acute relapses are managed with high-dose intravenous methylprednisolone, often combined with plasma exchange in severe attacks (EDSS ≥4 or visual acuity <0.1). For maintenance therapy, azathioprine or mycophenolate mofetil, either as monotherapy or combined with corticosteroid, is recommended. More recently, monoclonal antibodies including inebilizumab and satralizumab have been reimbursed in Taiwan under strict National Health Insurance regulations, restricted to AQP4-IgG-seropositive patients with high disease severity refractory to oral immunotherapy. This review provides an updated overview of NMOSD diagnosis and treatment, with emphasis on the characteristics of Taiwanese patients."

Journal • Review • CNS Disorders • Hepatitis B • Immunology • Infectious Disease • Inflammation • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases