Johnson & Johnson seeks first approval of nipocalimab to treat broadest population living with antibody positive generalized myasthenia gravis
Johnson & Johnson (NYSE: JNJ) has submitted a Biologics License Application to the FDA for nipocalimab, seeking its first global approval to treat generalized myasthenia gravis (gMG). The application is based on the Phase 3 Vivacity-MG3 study, which demonstrated superior outcomes for antibody-positive participants receiving nipocalimab plus standard of care compared to placebo. The study included anti-AChR+, anti-MuSK+, and anti-LRP4+ antibody positive adults, covering about 95% of the gMG patient population.
Nipocalimab is the first FcRn blocker to show sustained disease control over 24 weeks with consistent dosing every other week. Its high binding affinity and specificity to FcRn's IgG binding site potentially differentiate it in the FcRn blocker class. The drug's safety and tolerability were consistent with previous studies.
Johnson & Johnson (NYSE: JNJ) ha presentato una Domanda di Licenza Biologica alla FDA per nipocalimab, cercando la sua prima approvazione globale per il trattamento della miastenia gravis generalizzata (gMG). La domanda si basa sullo studio di Fase 3 Vivacity-MG3, che ha dimostrato risultati superiori per i partecipanti positivi agli anticorpi che ricevevano nipocalimab più il trattamento standard rispetto al placebo. Lo studio includeva adulti positivi agli anticorpi anti-AChR+, anti-MuSK+ e anti-LRP4+, coprendo circa il 95% della popolazione di pazienti affetti da gMG.
Nipocalimab è il primo inibitore di FcRn a mostrare un controllo della malattia sostenuto per 24 settimane con una somministrazione costante ogni due settimane. La sua alta affinità di legame e specificità per il sito di legame dell'IgG di FcRn potrebbero distinguerlo nella classe dei bloccanti di FcRn. La sicurezza e la tollerabilità del farmaco sono state coerenti con studi precedenti.
Johnson & Johnson (NYSE: JNJ) ha presentado una Solicitud de Licencia Biológica a la FDA para nipocalimab, buscando su primera aprobación global para tratar la miastenia gravis generalizada (gMG). La solicitud se basa en el estudio de Fase 3 Vivacity-MG3, que demostró resultados superiores para los participantes positivos a anticuerpos que recibieron nipocalimab junto con el tratamiento estándar en comparación con el placebo. El estudio incluyó a adultos positivos a anticuerpos anti-AChR+, anti-MuSK+ y anti-LRP4+, abarcando aproximadamente el 95% de la población de pacientes con gMG.
Nipocalimab es el primer bloqueador de FcRn que muestra un control sostenido de la enfermedad durante 24 semanas con dosificación constante cada dos semanas. Su alta afinidad de unión y especificidad para el sitio de unión de IgG de FcRn pueden diferenciarlo en la clase de bloqueadores de FcRn. La seguridad y tolerabilidad del medicamento eran consistentes con estudios previos.
존슨앤드존슨(NYSE: JNJ)는 생물학적 제품 허가 신청(BLA)을 FDA에 제출하여 니포칼리맙의 첫 번째 글로벌 승인을 얻으려고 합니다. 이는 일반화된 근무력증(gMG) 치료를 위한 것입니다. 이 신청서는 3상 비바시티-MG3 연구를 기반으로 하며, 연구에서는 니포칼리맙과 표준 치료를 받는 항체 양성 참여자들이 플라시보에 비해 우수한 결과를 보였음을 입증했습니다. 연구에는 항체 양성 성인인 anti-AChR+, anti-MuSK+ 및 anti-LRP4+가 포함되어 있으며, 이는 gMG 환자 인구의 약 95%를 포함하고 있습니다.
니포칼리맙은 24주 동안 지속적인 질병 조절을 보여준 첫 번째 FcRn 차단제이며, 격주로 일정하게 투여됩니다. FcRn의 IgG 결합 부위에 대한 높은 결합 친화성과 특이성은 FcRn 차단제 클래스에서의 차별성을 나타낼 수 있습니다. 약물의 안전성과 내약성은 이전 연구와 일관성이 있었습니다.
Johnson & Johnson (NYSE: JNJ) a soumis une Demande de Licence Biologique à la FDA pour nipocalimab, cherchant à obtenir sa première approbation mondiale pour traiter la myasthénie grave généralisée (gMG). La demande est basée sur l'étude de Phase 3 Vivacity-MG3, qui a démontré des résultats supérieurs pour les participants positifs aux anticorps recevant nipocalimab associé aux soins standard par rapport à un placebo. L'étude a inclus des adultes positifs aux anticorps anti-AChR+, anti-MuSK+ et anti-LRP4+, couvrant environ 95% de la population de patients gMG.
Nipocalimab est le premier bloqueur de FcRn à montrer un contrôle durable de la maladie pendant 24 semaines avec une posologie régulière toutes les deux semaines. Sa forte affinité de liaison et sa spécificité pour le site de liaison IgG de FcRn pourraient le distinguer dans la classe des bloqueurs de FcRn. La sécurité et la tolérance du médicament étaient cohérentes avec les études précédentes.
Johnson & Johnson (NYSE: JNJ) hat einen Biologischen Lizenzantrag bei der FDA für Nipocalimab eingereicht und sucht die erste globale Zulassung zur Behandlung von generalisierten Myasthenia gravis (gMG). Der Antrag basiert auf der Phase-3-Studie Vivacity-MG3, die überlegene Ergebnisse für antibody-positive Teilnehmer zeigte, die Nipocalimab in Kombination mit der Standardbehandlung im Vergleich zu Placebo erhielten. Die Studie umfasste anti-AChR+, anti-MuSK+ und anti-LRP4+ antibody-positive Erwachsene, die etwa 95% der gMG-Patientenpopulation abdeckten.
Nipocalimab ist der erste FcRn-Blocker, der über 24 Wochen nachhaltige Krankheitskontrolle mit konstanten Dosierungen alle zwei Wochen zeigt. Seine hohe Bindungsaffinität und Spezifität für die IgG-Bindungsstelle von FcRn könnten ihn in der Klasse der FcRn-Blocker differenzieren. Die Sicherheit und Verträglichkeit des Medikaments waren konsistent mit früheren Studien.
- First FDA submission for nipocalimab, potentially expanding treatment options for gMG patients
- Phase 3 Vivacity-MG3 study showed superior outcomes for nipocalimab plus standard of care vs placebo
- Nipocalimab demonstrated sustained disease control over 24 weeks, the longest period assessed for an FcRn blocker in gMG
- Study included the broadest population of antibody-positive gMG patients (95% of patient population)
- High binding affinity and specificity to FcRn's IgG binding site may differentiate nipocalimab in its class
- None.
Insights
Johnson & Johnson's submission of a Biologics License Application (BLA) for nipocalimab marks a significant milestone in the treatment of generalized myasthenia gravis (gMG). The Phase 3 Vivacity-MG3 study results are particularly noteworthy, demonstrating sustained disease control over 24 weeks in a broad population of antibody-positive patients. This is important as it covers
The primary endpoint, measuring improvement in MG-ADL score, showed superior outcomes for nipocalimab plus standard of care compared to placebo. This consistent efficacy over six months of dosing is unprecedented in the FcRn blocker class for gMG. The high binding affinity and specificity of nipocalimab to FcRn could potentially differentiate it from other treatments, offering a new therapeutic option for this chronic, life-long disease.
This BLA submission could significantly impact Johnson & Johnson's market position in the neuroscience and immunology sectors. If approved, nipocalimab would be the first in its class for treating such a broad gMG population, potentially capturing a substantial market share. The drug's unique molecular properties and efficacy profile could lead to strong market adoption and revenue growth.
Investors should note that this expands J&J's portfolio in high-value, chronic disease treatments, which typically offer stable, long-term revenue streams. However, the approval process and potential market launch timelines are important factors to monitor. Competition in the gMG space and pricing strategies will also play key roles in determining the financial impact of nipocalimab on J&J's bottom line.
Marks first FDA submission for nipocalimab, an investigational treatment that binds with high affinity and specificity to block FcRn and reduce levels of autoantibodies
Filing based on the Phase 3 Vivacity-MG3 program, the first-and-only study results in the class demonstrating sustained disease control over 24 weeks in antibody positive adult patients: anti-AChR+, anti-MuSK+, anti-LRP4+
The application included data from the Phase 3 Vivacity-MG3 study which showed that outcomes for a broad population of antibody positive participants who received nipocalimab plus standard of care (SOC) were superior compared to those who received placebo plus SOC. The primary endpoint of the study measured improvement in the MG-ADLa score from baseline over 24 weeks and study participants included anti-AChR+, anti-MuSK+, and anti-LRP4+b antibody positive adults, which account for approximately 95 percent of the gMG patient population, making Vivacity-MG3 the first-and-only study to demonstrate sustained disease control in these subtypes.1,2 Safety and tolerability were consistent with other nipocalimab studies.3,4,5
"We are encouraged by the potential of nipocalimab to provide sustained disease control for people living with generalized myasthenia gravis, a chronic, life-long disease," said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Johnson & Johnson Innovative Medicine. "The filing for approval of nipocalimab represents an important step forward as Johnson & Johnson continues to push the boundaries of research to develop innovative solutions to treat autoantibody-driven diseases, building on decades of expertise in neuroscience and immunology. We look forward to working with the FDA in their review of the data supporting the submission."
Nipocalimab is the first-and-only FcRn blocker to demonstrate sustained disease control measured by improvement in MG-ADL when added to background SOC compared with placebo plus SOC over a period of six months of consistent dosing (every other week)c, which is the longest period of controlled safety and efficacy assessment of an FcRn blocker in gMG.
Earlier this year at the American Academy of Neurology Annual Meeting, Johnson & Johnson presented data focused on the molecular properties of nipocalimab. Characteristics such as its high binding affinity and specificity to the immunoglobulin G (IgG) binding site of FcRn have the potential to differentiate nipocalimab in the FcRn blocker class of treatments.6 These properties, along with the dosing regimen chosen for the study, are thought to lower IgG, including IgG autoantibodies in diseases such as gMG and other autoantibody-driven diseases.7
Editor's notes:
a. MG-ADL (Myasthenia Gravis – Activities of Daily Living) provides a rapid clinical assessment of the patient's recall of symptoms impacting activities of daily living, with a total score range of 0 to 24; a higher score indicates greater symptom severity.
b. Positive patients include anti-acetylcholine receptor positive antibody (AChR+), anti-muscle specific tyrosine kinase positive antibody (MuSK+) and/or anti-low density lipoprotein receptor-related protein 4 positive antibody (LRP4+).
c. Patients who received nipocalimab plus current SOC had a mean change of -4.70 [standard error (SE) 0.329]. Patients on placebo plus current SOC had a mean change of -3.25 (SE 0.335); difference of least-squares (LS) means -1.45 [0.470]; P=0.002.
About Generalized Myasthenia Gravis (gMG)
Myasthenia gravis (MG) is an autoantibody disease in which autoantibodies target proteins at the neuromuscular junction, disrupt neuromuscular signaling, and impair or prevent muscle contraction.8 In MG, the immune system mistakenly attacks proteins at the neuromuscular junction by producing antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-low density lipoprotein-related protein 4 [LRP4]) that can block or disrupt normal functioning, preventing signals from transferring from nerves to muscles.9 The disease impacts an estimated 700,000 people worldwide.8 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently impacts young women and older men.10 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential.11,12,13
Initial disease manifestations are usually ocular but in 85 percent or more14,15 cases, the disease generalizes (gMG), which is characterized by fluctuating weakness of the skeletal muscles leading to symptoms like limb weakness, drooping eyelids, double vision and difficulties with chewing, swallowing, speech, and breathing.8,16,17 Approximately 100,000 individuals in the
About the Phase 3 Vivacity-MG3 Study
The Phase 3 Vivacity-MG3 study was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic disease where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of which were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial. Randomization was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC. Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo). The primary endpoint of the study was mean change in MG-ADLa score from baseline over Weeks 22, 23 and 24 in antibody positive patients. A key secondary endpoint included change in Quantitative Myasthenia Gravis (QMG) score, which is a 13-item assessment by a clinician that quantifies MG disease severity. Long-term safety and efficacy were further assessed in an ongoing OLE phase.19
About Nipocalimab
Nipocalimab is an investigational monoclonal antibody, purposefully designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies, while preserving immune function without causing broad immunosuppression. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology.19,20,21,22,23,24,25,26,27 Blockade of IgG binding to FcRn in the placenta is also believed to prevent transplacental transfer of maternal alloantibodies to the fetus.28,29
The
U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024 by the FDAU.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 by the FDAU.S. FDA Breakthrough Therapy designation for HDFN in February 2024 by the FDA- EU EMA Orphan medicinal product designation for HDFN in October 2019 by the EMA
About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.
Learn more at https://www.jnj.com/ or at www.janssen.com/johnson-johnson-innovative-medicine.
Follow us at @JanssenUS and @JNJInnovMed.
Janssen Research & Development, LLC and Janssen Biotech, Inc. are both Johnson & Johnson companies.
Cautions Concerning Forward-Looking Statements
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
1 Antozzi, C et al., Efficacy and Safety of Nipocalimab in patients with Generalized Myasthenia Gravis- Top Line Results from the Double-Blind, Placebo-Controlled, Randomized Phase 3 Vivacity-MG3 study. 2024 European Academy of Neurology Congress. June 2024.
2 Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev Clin Immunol 2012; 8: 427–38
3 Kenneth J. Moise Jr., et al. Nipocalimab in Early-onset Severe Hemolytic Disease of the Fetus & Newborn. N Engl J Med. 2024; DOI: 10.1056/NEJMoa2314466.
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11 Ye, Yun et al. Epidemiology of myasthenia gravis in
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13 Johnson & Johnson Data on file.
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FAQ
What is the purpose of Johnson & Johnson's (JNJ) recent FDA submission for nipocalimab?
What were the key findings of the Phase 3 Vivacity-MG3 study for JNJ's nipocalimab?
How does nipocalimab (JNJ) potentially differentiate itself from other FcRn blockers?
What percentage of the gMG patient population was represented in JNJ's Vivacity-MG3 study for nipocalimab?
