Nipocalimab granted U.S. FDA Priority Review for the treatment of generalized myasthenia gravis

Rhea-AI Summary

Johnson & Johnson (NYSE: JNJ) announced that nipocalimab has received FDA Priority Review designation for treating antibody positive patients with generalized myasthenia gravis (gMG). The designation is supported by the Phase 3 Vivacity-MG3 study results, which showed sustained disease control over 24 weeks in patients with anti-AChR, anti-MuSK, and anti-LRP4 antibodies.

The study demonstrated that nipocalimab plus standard of care achieved a significantly greater reduction in MG-ADL response compared to placebo (p=0.0213). gMG affects approximately 700,000 people worldwide and currently has no cure. The company has also submitted a Marketing Authorisation Application to the EMA for nipocalimab in gMG on September 11, 2024.

Additionally, nipocalimab recently received FDA Breakthrough Therapy Designation for treating adults with moderate-to-severe Sjögren's disease, based on Phase 2 DAHLIAS study results.

Positive

• FDA Priority Review designation received, potentially expediting approval process
• Phase 3 trial demonstrated significant efficacy vs placebo (p=0.0213)
• Broad treatment potential covering multiple antibody types (anti-AChR, anti-MuSK, anti-LRP4)
• Large addressable market of 700,000 patients worldwide
• Additional potential in Sjögren's disease with Breakthrough Therapy Designation

Negative

• None.

Insights

Medical Research Analyst

The FDA's Priority Review for nipocalimab represents a significant regulatory milestone for Johnson & Johnson's autoimmune disease portfolio. The Phase 3 Vivacity-MG3 study data demonstrates efficacy across all three antibody subtypes (anti-AChR, anti-MuSK, anti-LRP4), which is particularly notable as most existing treatments don't show such broad coverage.

The MG-ADL improvement threshold of ≥2 points achieved in the trial (p=0.0213) is clinically meaningful, as it can translate to substantial quality-of-life improvements for patients. For context, this improvement could mean the difference between requiring ventilator support versus breathing independently. With an estimated 700,000 patients globally affected by gMG, the market potential is substantial.

The concurrent EMA submission and recent Breakthrough Therapy Designation for Sjögren's disease suggest a robust development pipeline that could significantly expand nipocalimab's commercial potential beyond gMG.

Financial Analyst

This regulatory advancement could substantially impact JNJ's revenue potential in the rare disease space. The global gMG therapeutics market is projected to reach $3.7 billion by 2027 and nipocalimab's broad antibody coverage could capture a significant market share. The Priority Review status accelerates the potential time to market by approximately 4 months compared to standard review.

Key competitors in this space include Argenx's Vyvgart and UCB's Rystiggo, but nipocalimab's potential advantage in treating all three antibody subtypes could differentiate it in the market. The additional Breakthrough Therapy Designation for Sjögren's disease further enhances the drug's commercial prospects, potentially creating multiple revenue streams from a single asset.

For investors, this development strengthens JNJ's position in the high-margin rare disease segment and could provide meaningful revenue growth in the medium term.

Biologics License Application acceptance supported by results from the Phase 3 Vivacity-MG3 study

Results demonstrate sustained disease control over 24 weeks in a broad population of antibody positive adult patients: anti-AChR, anti-MuSK, anti-LRP4

SPRING HOUSE, Pa., Jan. 9, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced the nipocalimab Biologics License Application (BLA) received Priority Review designation from the U.S Food and Drug Administration (FDA) for the treatment of antibody positive (anti-AChR, anti-MuSK, anti-LRP4) patients with generalized myasthenia gravis (gMG), as supported by findings from the Phase 3 Vivacity-MG3 study. The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.¹

"We welcome the FDA's decision to grant Priority Review for the treatment of generalized myasthenia gravis, which underscores the need for additional treatment options in a broad population of people living with gMG," said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader at Johnson & Johnson Innovative Medicine. "We are committed to working closely with the FDA to help bring nipocalimab as a potential treatment to certain patients living with gMG, and we especially thank the participants in the Phase 2 and 3 studies. If approved, nipocalimab has the potential to treat gMG in antibody positive individuals, including anti-AChR, anti-MuSK, and/or anti-LRP4."

gMG is a chronic, life-long, rare, autoantibody-driven disease, for which no cure is currently available.^2,3 gMG impacts an estimated 700,000 people worldwide.^2,3 In the Phase 3 study, nipocalimab plus standard of care (SOC) demonstrated a significantly greater reduction in MG-ADL response (≥2-point improvement from baseline) compared with placebo plus SOC (p=0.0213).⁴ For someone living with gMG, a 1- to 2-point change on MG-ADL may be the difference between normal eating and frequent choking on food, or shortness of breath at rest and being on a ventilator.⁵  

Johnson & Johnson also submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of nipocalimab in gMG on September 11, 2024.⁶ In addition, nipocalimab recently received U.S. FDA Breakthrough Therapy Designation for the treatment of adults with moderate-to-severe Sjögren's disease as supported by results from the Phase 2 DAHLIAS study.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.5

ABOUT GENERALIZED MYASTHENIA GRAVIS (gMG)

Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-low density lipoprotein-related protein 4 [LRP4]), which target proteins at the neuromuscular junction and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction.^2,8,9 The disease impacts an estimated 700,000 people worldwide.² Approximately 10 to 15% of new cases of MG are diagnosed in adolescents (12 – 17 years of age).^10,11,12 Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the US diagnosed in girls.^13,14,15

Initial disease manifestations are usually ocular but in 85% or more 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.^16,17,18,19 Approximately 100,000 individuals in the U.S. are living with gMG.²⁰ Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.²¹ Currently, SOC treatments for adolescents with gMG are extrapolated from adult trials.¹² Other than symptomatic treatments, there are no approved FcRn blockers for adolescents with gMG in the United States.¹²  

ABOUT THE PHASE 3 VIVACITY-MG3 STUDY

The Phase 3 Vivacity-MG3 study (NCT04951622) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing standard of care (SOC) therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.^4,22 Randomisation 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.⁴ Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo).⁴ The primary endpoint of the study was mean change in MG-ADL^a score from baseline over Weeks 22, 23 and 24 in antibody positive patients. A key secondary endpoint included change in QMG score. Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase.²²

ABOUT NIPOCALIMAB 

Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. 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 Rheumatology.^{22, 23, 24,25,26,27,28,29,30} Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.^31,32

The FDA and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:  

• 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
• U.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
• U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren's disease in November 2024
• U.S. FDA granted Priority Review in gMG in Q4 2024
• EU EMA Orphan medicinal product designation for HDFN in October 2019

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.innovativemedicine.jnj.com

Follow us at @JNJInnovMed.

Janssen Research & Development, LLC and Janssen Biotech, Inc. are 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.

References

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Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. 6 Johnson & Johnson EMEA. Johnson & Johnson seeks first EU approval of nipocalimab to treat a broad population of patients living with antibody-positive generalised myasthenia gravis. Available at: https://innovativemedicine.jnj.com/emea/newsroom/immunology/johnson-johnson-seeks-first-eu-approval-of-nipocalimab-to-treat-a-broad-population-of-patients-living-with-antibody-positive-generalised-myasthenia-gravis. Last accessed: November 2024. 7 Johnson & Johnson. Nipocalimab is the first and only investigational therapy granted U.S. FDA Breakthrough Therapy Designation for the treatment of adults living with moderate-to-severe Sjögren's disease. Available at: https://www.jnj.com/media-center/press-releases/nipocalimab-is-the-first-and-only-investigational-therapy-granted-u-s-fda-breakthrough-therapy-designation-for-the-treatment-of-adults-living-with-moderate-to-severe-sjogrens-disease. 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Gender prevalence in childhood multiple sclerosis and myasthenia gravis. J Child Neurol. 2002 May;17(5):390-2. doi: 10.1177/088307380201700516.  14 Parr JR, Andrew MJ, Finnis M, Beeson D, Vincent A, Jayawant S. How common is childhood myasthenia? The UK incidence and prevalence of autoimmune and congenital myasthenia. Arch Dis Child. 2014 Jun;99(6):539-42. doi: 10.1136/archdischild-2013-304788. 15 Mansukhani SA, Bothun ED, Diehl NN, Mohney BG. Incidence and Ocular Features of Pediatric Myasthenias. Am J Ophthalmol. 2019 Apr;200:242-249. doi: 10.1016/j.ajo.2019.01.004. 16 Bever, C.T., Jr, Aquino, A.V., Penn, A.S., Lovelace, R.E. and Rowland, L.P. (1983), Prognosis of ocular myasthenia. Ann Neurol., 14: 516-519. https://doi.org/10.1002/ana.410140504 17 Kupersmith MJ, Latkany R, Homel P. Development of generalized disease at 2 years in patients with ocular myasthenia gravis. Arch Neurol. 2003 Feb;60(2):243-8. doi: 10.1001/archneur.60.2.243. 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Media contact: Bridget Kimmel Mobile: (215) 688-6033           bkimmel@its.jnj.com

Investor contact: Lauren Johnson investor-relations@its.jnj.com

 

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SOURCE Johnson & Johnson

FAQ

What did the FDA grant JNJ's nipocalimab for generalized myasthenia gravis treatment?

The FDA granted Priority Review designation for JNJ's nipocalimab for the treatment of antibody positive patients with generalized myasthenia gravis (gMG).

What were the results of JNJ's Phase 3 Vivacity-MG3 study for nipocalimab?

The Phase 3 study showed that nipocalimab plus standard of care achieved a significantly greater reduction in MG-ADL response compared to placebo (p=0.0213), demonstrating sustained disease control over 24 weeks.

How many patients could potentially benefit from JNJ's nipocalimab for gMG?

Approximately 700,000 people worldwide are affected by generalized myasthenia gravis (gMG) and could potentially benefit from nipocalimab treatment.

When did JNJ submit the Marketing Authorisation Application to EMA for nipocalimab?

Johnson & Johnson submitted the Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for nipocalimab in gMG on September 11, 2024.

What additional indication has JNJ's nipocalimab received Breakthrough Therapy Designation for?

Nipocalimab received FDA Breakthrough Therapy Designation for the treatment of adults with moderate-to-severe Sjögren's disease.