Imaavy (nipocalimab-aahu) / J&J - LARVOL DELTA

Management of Autoimmune Hemolytic Anemia (ASH 2025) - "For relapsed/refractory patients rituximab has become the preferred second line-therapy, comparing favorably with the traditional splenectomy, which has been progressively abandoned or moved to further lines along with classic immunosuppressors. Several novel treatments are in development for wAIHA, encompassing drugs targeting B-cells (parsaclisib, ibrutinib, rilzabrutinib, zanubrutinib, obexelimab, ianalumab, povetacicept), plasma cells (bortezomib, daratumumab), spleen tyrosine kinase (fostamatinib, sovleplenib), and the neonatal Fc receptor (nipocalimab)."

IO biomarker • Anemia • Autoimmune Hemolytic Anemia • Bone Marrow Transplantation • Hematological Disorders • Immunology • Infectious Disease • HP • SYK

Nipocalimab: a significant milestone in the treatment of myasthenia gravis approved by the FDA. (PubMed, Ann Med Surg (Lond)) - "Late-stage trials have confirmed significant improvements in key health parameters, reinforcing the agent's therapeutic value. This biology introduces a more precise, well-tolerated, and durable option for treating myasthenia gravis."

FDA event • Journal • CNS Disorders • Immunology • Myasthenia Gravis

Management of autoimmune hemolytic anemia. (PubMed, Hematology Am Soc Hematol Educ Program) - "Rituximab is now the preferred second-line option for relapsed/refractory patients, comparing favorably with the traditional splenectomy. The latter is increasingly reserved for later lines together with classic immunosuppressants. Several novel treatments are in development for refractory wAIHA, encompassing drugs targeting B-cells (parsaclisib, ibrutinib, rilzabrutinib, zanubrutinib, obexelimab, ianalumab, povetacicept), plasma cells (bortezomib, daratumumab), spleen tyrosine kinase (fostamatinib, sovleplenib), and the neonatal Fc receptor (nipocalimab)."

Journal • Review • Anemia • Autoimmune Hemolytic Anemia • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Infectious Disease • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Transplantation • SYK

EPIC: Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=115 | Recruiting | Sponsor: Janssen Research & Development, LLC | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

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

Johnson & Johnson receives European Commission approval of IMAAVY (nipocalimab), a new FcRn blocker offering sustained disease control in a broad population of people living with generalised myasthenia gravis (gMG) (GlobeNewswire) - "This approval is for the treatment of a broad population of people living with gMG including adults and adolescent patients 12 years of age and older who are anti-acetylcholine receptor [AChR] or anti-muscle-specific kinase [MuSK] antibody-positive....This approval is supported by the data from the pivotal, ongoing Phase 3 Vivacity-MG3 study....This approval also includes data from the Phase 2/3 Vibrance-MG study of nipocalimab in anti-AChR antibody-positive adolescents (aged 12–17 years) living with gMG."

EMA approval • Myasthenia Gravis

Design of a Phase 3, Multicenter, Randomized, Open-label Study of Nipocalimab or IVIG and Prednisone in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia. (PubMed, Am J Perinatol) - "Exploratory endpoints include patient- and caregiver-reported outcome assessments and nipocalimab pharmacokinetics and pharmacodynamics. FREESIA-3, an open-label, multicenter, randomized, phase 3 study, will evaluate the efficacy and safety of nipocalimab in both standard-risk and high-risk pregnancies for FNAIT."

Clinical • Journal • P3 data • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Nipocalimab reduces Sjögren disease activity in phase II trial. (PubMed, Nat Rev Rheumatol) - No abstract available

Journal • P2 data • Sjogren's Syndrome

Nipocalimab for Sjögren's disease-the importance of autoantibodies. (PubMed, Lancet) - No abstract available

Journal • Sjogren's Syndrome

Efficacy and safety of nipocalimab in patients with moderate-to-severe Sjögren's disease (DAHLIAS): a randomised, phase 2, placebo-controlled, double-blind trial. (PubMed, Lancet) - P2 | "Fc receptor blockade by nipocalimab 15 mg/kg significantly improved clinical disease activity versus placebo and was safe and well tolerated in participants with moderate-to-severe, active Sjögren's disease. Reductions in IgG autoantibodies during nipocalimab treatment support their contribution to Sjögren's disease pathogenesis."

Clinical • Journal • P2 data • Fatigue • Immunology • Pain • Rheumatology • Sjogren's Syndrome

Design of a Phase 3, Multicenter, Randomized, Open-Label Study of Nipocalimab or IVIG in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia (FREESIA-3) (ASH 2024) - P2, P3 | "The open-label FREESIA-3 study aims to evaluate the efficacy and safety of nipocalimab with a contemporaneous reference arm of IVIG with/without prednisone in pregnancies at risk for FNAIT (ClinicalTrials.gov identifier : NCT06533098). Additional endpoints consist of safety, patient- and caregiver-reported outcomes, pharmacokinetics, pharmacodynamics, and immunogenicity of nipocalimab. Conclusion : The open-label FREESIA-3 trial will assess the efficacy and safety of nipocalimab in at-risk FNAIT pregnancies."

Clinical • P3 data • Anemia • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Perinatal Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Structure of the human astrovirus capsid spike in complex with the neonatal Fc receptor. (PubMed, Nat Commun) - "Additionally, we demonstrate that the FcRn inhibitor, nipocalimab, and anti-HAstV neutralizing monoclonal antibodies block HAstV spike binding to FcRn, revealing their neutralization mechanisms and supporting their therapeutic potential. Overall, our findings illuminate a crucial interaction in the HAstV life cycle, which may help to inform the development of a HAstV vaccine and antibody therapies."

Journal • Gastroenterology • Gastrointestinal Disorder

Biologics in red cell and platelet alloimmunizations: State of the science and future perspectives. (PubMed, Best Pract Res Clin Obstet Gynaecol) - "IVIG with or without oral steroids is utilized in most countries to prevent thrombocytopenia and intracranial hemorrhage in cases of fetal/neonatal alloimmune thrombocytopenia (FNAIT). Nipocalimab, a humanized monoclonal antibody that blocks the neonatal Fc receptor, is currently undergoing clinical trials in both HDFN and FNAIT as a potential new form of immunotherapy for these alloimmune disorders of pregnancy."

Journal • Review • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Vibrance Pediatric Phase 2/3 Study (PRNewswire) - "The announcement of the EPIC study is accompanied by new positive data from the Vibrance-MG Phase 2/3 LTE study. These new data show treatment with IMAAVY plus standard of care in pediatric patients aged 12 and older with gMG demonstrated sustained reductions in IgG through 72 weeks, disease control, functional improvements and long-term safety...New data show that treatment with IMAAVY led to a rapid and sustained reduction in IgG levels – with a median reduction of ~73% by Week 24. Improvements in both daily functionc and muscle strengthd were also observed through 72 weeks, indicating sustained disease control over time. The treatment was generally well tolerated and no new safety concerns have emerged thus far during extended follow-up."

P2/3 data • Myasthenia Gravis

Johnson & Johnson announces first head-to-head study comparing IMAAVY with an alternative FcRn blocker in generalized myasthenia gravis (gMG) at AANEM Annual Meeting (PRNewswire) - "The EPIC study design...are among 38 abstracts the Company will present at the 2025 Myasthenia Gravis Foundation of America (MGFA) Scientific Session..."

Clinical data • Clinical protocol • Myasthenia Gravis

Clinically Relevant Anti-Vaccine and Virus Antibodies in Patients with Sjogren's Disease Treated with Nipocalimab: Post-Hoc Analysis of the DAHLIAS Study (ACR Convergence 2025) - P2 | "In nipocalimab-treated participants, preexisting anti-TT and anti-VZV antibodies followed a similar pattern as total IgG, consistent with its mechanism of action. Additionally, nipocalimab did not prevent humoral responses to SARS-CoV-2 infection or vaccination in participants with Sjögren's Disease in the DAHLIAS study. These observations suggest that patients treated with nipocalimab can follow recommended vaccination schedules concomitant with nipocalimab treatment as appropriate.Cossu M, et al."

Clinical • Retrospective data • Herpes Zoster • Immunology • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • Rheumatology • Sjogren's Syndrome • Tetanus • Varicella Zoster

From promise to practice: evaluating the clinical impact of FcRn inhibition in IgG-mediated autoimmune rheumatic diseases. (PubMed, Front Immunol) - "Several FcRn-targeting agents, such as efgartigimod, rozanolixizumab, and nipocalimab, have demonstrated favorable safety and efficacy profiles in clinical trials and are now approved or under investigation for multiple indications. This review also explored personalized therapeutic approaches, combination strategies, and the future landscape of FcRn-targeted drug development. While FcRn inhibition offered a paradigm shift in managing antibody-driven diseases, long-term safety and patient stratification remain key challenges for future research."

Journal • Review • Immunology • Inflammatory Arthritis • Lupus • Rheumatoid Arthritis • Rheumatology • Systemic Lupus Erythematosus • Vasculitis

EPIC: Comparative Efficacy of Nipocalimab and Efgartigimod in Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=115 | Not yet recruiting | Sponsor: Janssen Research & Development, LLC

New P3 trial • CNS Disorders • Myasthenia Gravis

DAFFODIL: Nipocalimab in Moderate to Severe Sjogren's Disease (clinicaltrials.gov) - P3 | N=600 | Recruiting | Sponsor: Janssen Research & Development, LLC | Trial completion date: Apr 2030 ➔ Aug 2029

Trial completion date • Immunology • Sjogren's Syndrome

THE ROLE OF FCRN ANTAGONISTS IN TRANSFORMING THE MYASTHENIA GRAVIS TREATMENT PARADIGM (WCN 2025) - "Early clinical trials of FcRn antagonists, such as efgartigimod, rozanolixizumab, and nipocalimab, have shown significant potential, including the reduction of symptom severity, rapid clinical improvement, and enhanced quality of life for patients with generalized myasthenia gravis (gMG). Furthermore, a Phase III clinical trial conducted in China for another FcRn antagonist, batoclimab, has also demonstrated positive results, further confirming the effectiveness and safety of this class of medications. The advent of FcRn antagonists marks a significant milestone in the management of MG, opening a new chapter of precision therapy in autoimmune neurology and offering a promising treatment paradigm for other IgG-mediated diseases."

CNS Disorders • Myasthenia Gravis

An evaluation of nipocalimab for the treatment of generalized myasthenia gravis. (PubMed, Expert Opin Biol Ther) - P2, P3 | "Clinical studies have demonstrated that nipocalimab provided rapid and sustained reduction of total IgG and pathogenic IgG autoantibodies together with sustained disease control over 6 months in a broad population of seropositive patients with gMG, with an acceptable safety profile. The long-term impact of nipocalimab on the course of gMG needs to be further investigated in real-world settings."

Journal • Review • CNS Disorders • Immunology • Myasthenia Gravis

Vibrance-MG: A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis (clinicaltrials.gov) - P2/3 | N=12 | Recruiting | Sponsor: Janssen Research & Development, LLC | Trial completion date: Dec 2026 ➔ Jun 2026

Trial completion date • CNS Disorders • Myasthenia Gravis

On 18 September 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Imaavy, intended for the treatment of generalised myasthenia gravis. (European Medicines Agency) - "Imaavy is indicated as an add-on to standard therapy for the treatment of generalised Myasthenia Gravis (gMG) in adult and adolescent patients aged 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive."

CHMP • Myasthenia Gravis

Nipocalimab Dose Selection in Generalized Myasthenia Gravis. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "The PK of nipocalimab and its effect on IgG reduction were similar between healthy participants and participants with gMG. Model-based simulations indicated that the IV dose of 15 mg/kg Q2W, starting 2 weeks after a 30 mg/kg IV loading dose, was the lowest Q2W maintenance dose predicted to achieve the target of 70% median of the average change in IgG reduction in participants with gMG and was the recommended dose for the pivotal phase 3 Vivacity-MG3 study in a gMG population."

Journal • CNS Disorders • Dyslipidemia • Myasthenia Gravis

Future directions in the management of warm autoimmune hemolytic anemia (PubMed, Rinsho Ketsueki) - "Rituximab and splenectomy are effective second-line treatments; however, there is no consensus on the optimal therapeutic approach for patients with wAIHA that is refractory to second-line therapy...Nipocalimab, a fully human IgG1 monoclonal antibody targeting FcRn, is expected to be effective in wAIHA by reducing the half-life of pathogenic IgG autoantibodies. Fostamatinib, an SyK inhibitor approved for the treatment of chronic immune thrombocytopenia in adults, demonstrated a significantly higher rate of sustained hemoglobin responses compared to placebo in the global, randomized, double-blind, placebo-controlled phase 3 FORWARD trial. The efficacy of Sovleplenib, another SyK inhibitor, was also demonstrated in a randomized, double-blind, placebo-controlled phase 2 trial."

Clinical • Journal • Review • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • SYK