Nipocalimab, the first and only investigational treatment to be granted U.S. FDA Breakthrough Therapy designation for the treatment of adults with moderate-to-severe Sjögren's disease, has now received Fast Track designation

Rhea-AI Summary

Johnson & Johnson (NYSE: JNJ) announced that nipocalimab has received Fast Track designation (FTD) from the FDA for treating adult patients with moderate-to-severe Sjögren's disease (SjD). This follows its previous Breakthrough Therapy designation (BTD), making it the first and only therapeutic to receive both designations for SjD treatment.

The Phase 2 DAHLIAS study showed promising results with nipocalimab demonstrating:

• Over 70% relative average improvement in systemic disease activity at Week 24 compared to placebo
• IgG reductions exceeding 77%
• Positive trends across multiple secondary endpoints
• Safety and tolerability consistent with other nipocalimab studies

Currently, there are no FDA-approved advanced treatments for Sjögren's disease, which can cause chronic dryness of moisture-producing glands and lead to systemic complications, including a 20 times greater risk of developing B-cell lymphomas compared to the general population.

Positive

• First and only treatment to receive both FDA Fast Track and Breakthrough Therapy designations for Sjögren's disease
• Strong Phase 2 efficacy data showing >70% disease activity improvement
• Significant IgG reductions of >77% in clinical trials
• Favorable safety and tolerability profile consistent with previous studies
• Addresses large unmet medical need with no current FDA-approved advanced treatments

Negative

• Still in clinical development phase with no guaranteed FDA approval
• Phase 3 trial results pending and still enrolling patients

Insights

Financial Analyst

Johnson & Johnson's nipocalimab receiving FDA Fast Track designation for Sjögren's disease represents significant regulatory advancement that could accelerate the drug's path to commercialization. This designation, combined with the previously granted Breakthrough Therapy designation, enhances the likelihood of priority review and potential expedited approval.

The commercial opportunity is substantial considering no advanced therapies are currently approved for Sjögren's disease, positioning J&J to potentially capture an untapped market with significant unmet medical needs. The addressable market is considerable given Sjögren's prevalence as a common autoimmune disorder.

The Phase 2 DAHLIAS study results showing 70% relative improvement in disease activity and 77% IgG reductions suggest strong efficacy which, if replicated in the ongoing Phase 3 DAFFODIL trial, would strengthen nipocalimab's value proposition. This represents the fourth Fast Track designation for nipocalimab across different indications, indicating the drug's potential as a significant growth driver across multiple autoimmune conditions.

This regulatory milestone strengthens J&J's immunology portfolio, potentially offsetting pressures in other segments and supporting the company's strategic focus on innovative medicine for complex diseases with treatment options.

Pharmaceutical Regulatory Expert

The FDA's decision to grant Fast Track designation (FTD) to nipocalimab for Sjögren's disease, following its Breakthrough Therapy designation (BTD), represents a significant regulatory accomplishment that substantially enhances the drug's development trajectory. These dual designations are relatively rare and reflect the FDA's recognition of both the serious nature of Sjögren's disease and the promising clinical evidence from nipocalimab's Phase 2 DAHLIAS trial.

The regulatory implications are substantial: FTD enables more frequent FDA interactions, Rolling Review submission potential, and possible Accelerated Approval and Priority Review eligibility. Combined with BTD, which offers intensive FDA guidance and organizational commitment, these designations create an optimized regulatory pathway with potentially shortened approval timelines.

Johnson & Johnson now has a clear regulatory advantage with nipocalimab being the only therapy with BTD for Sjögren's disease. The Phase 2 efficacy data showing 70% improvement versus placebo provides robust evidence supporting these designations. With the Phase 3 DAFFODIL study actively enrolling, J&J is positioned to potentially file for approval with enhanced regulatory support assuming positive outcomes.

This represents nipocalimab's fourth FTD across different indications, suggesting the FDA recognizes the broad therapeutic potential of this FcRn blocker mechanism across multiple autoimmune conditions, strengthening the overall regulatory position of this asset.

Sjögren's disease (SjD) is a prevalent, debilitating autoantibody disease with no FDA-approved advanced treatments  

The Company is actively enrolling patients in the Phase 3 DAFFODIL study  

This marks the fourth nipocalimab FDA Fast Track designation

SPRING HOUSE, Pa., March 18, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced that the U.S. Food and Drug Administration (FDA) has granted investigational nipocalimab Fast Track designation (FTD) for the treatment of adult patients with moderate-to-severe Sjögren's disease (SjD), having previously been granted Breakthrough Therapy designation (BTD) for the investigational therapy late last year. Currently, no advanced therapies are approved to treat this disease.

Building upon the BTD, which nipocalimab is the first and only therapeutic to receive for SjD, the U.S. FDA's FTD is also designed to accelerate the delivery of new therapeutics to patients by facilitating the development and expediting the review of drugs that demonstrate the potential to treat serious conditions and help address unmet needs for serious or life-threatening conditions.

"This marks an additional important step forward in our efforts to bring meaningful advancements to people living with Sjögren's disease, a serious and debilitating condition. We look forward to continuing to work closely with the FDA to advance the clinical development of nipocalimab and potentially provide a much-needed treatment option for this community," said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Disease Area Leader, Johnson & Johnson Innovative Medicine.

There are no FDA-approved treatments that directly address the underlying causes of this complex disease, associated with serious health consequences including chronic dryness of moisture producing glands that may lead to systemic complications such as joint pain, fatigue and inflammation in multiple organ systems.^1,2,3 These systemic complications can lead to an increased risk of mortality and associated health conditions, including a 20 times greater risk of developing B-cell lymphomas when compared to the general population.^1,4 

The Phase 2 DAHLIAS study, the results of which were presented last year, represented the first-ever positive results of an investigational FcRn blocker as a potential targeted therapy in SjD. The study achieved the primary endpoint in the 15 mg/kg Q2W nipocalimab group, showing a greater than 70% relative average improvement in systemic disease activity at Week 24 compared to placebo and IgG reductions of more than 77%.⁵ Trends of improvement were similarly observed across multiple secondary endpoints.⁵ Safety and tolerability were consistent with other nipocalimab clinical studies.⁵

ABOUT SJÖGREN'S DISEASE

Sjögren's disease (SjD) is one of the most prevalent autoantibody-driven diseases for which no therapies are currently approved that treat the underlying and systemic nature of the disease.¹ It is a chronic autoimmune disease that is estimated to impact approximately four million people worldwide and is nine times more common in women than men.^6,7 SjD is characterized by autoantibody production, chronic inflammation, and lymphocytic infiltration of exocrine glands. Most patients are affected by mucosal dryness (eyes, mouth, vagina), joint pain and fatigue.¹ More than 50% of SjD patients have a moderate to severe form of the condition, and disease burden can be as high as that of rheumatoid arthritis or systemic lupus erythematosus. It is usually associated with impaired quality of life and functional capacity.^6.8,9 

ABOUT DAHLIAS

DAHLIAS (NCT04968912) is a Phase 2 multicenter, randomized, placebo-controlled double-blind study to evaluate the effects of nipocalimab in participants with primary Sjögren's disease. DAHLIAS is a Phase 2 dose-ranging study for adults with moderately-to-severely active primary SjD who were seropositive for anti-Ro60 and/or anti-Ro52 IgG antibodies. 163 adults aged 18-75 were randomized 1:1:1 to receive intravenous nipocalimab at 5 or 15 mg/kg or placebo every 2 weeks through Week 22 and received protocol-permitted background standard of care. Safety assessments were conducted through Week 30. The primary endpoint was change in baseline in the ClinESSDAI (Clinical European League Against Rheumatism Sjögren's Syndrome Disease Activity Index) Score at Week 24. ClinESSDAI is a systemic diseases activity index designed to measure disease activity in patients with primary SjD based on 11 domains including: constitutional, lymphadenopathy, glandular, articular, cutaneous, respiratory, renal, muscular, peripheral nervous system, central nervous system, and hematological. 

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 Rheumatic diseases.^{5,10,11,12,13,14,15,16.17} Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.^18,19

The U.S. Food and Drug Administration (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 and Sjögren’s disease (SjD) in March 2025
• 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 https://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 most recent Annual Report on Form 10-K, 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

1	Huang H, Xie W, Geng Y, Fan Y, Zhang Z. Mortality in patients with primary Sjögren's syndrome: a systematic review and meta-analysis. Rheumatology (Oxford). 2021 Sep 1;60(9):4029-4038. doi: 10.1093/rheumatology/keab364. PMID: 33878179.
2	Sjogren's Disease Foundation. Understanding Sjogrens – Treatment. Available at: https://sjogrens.org/. Last accessed: March 2025.
3	Mayo Clinic. Sjogren's syndrome. Available at: https://www.mayoclinic.org/diseases-conditions/sjogrens-syndrome/symptoms-causes/syc-20353216. Last accessed: March 2025.
4	Brito-Zeron, P., Flores-Chavez, A., Horvath, Fanny I., Rasmussen, A., et al. Mortality risk factors in primary Sjogren syndrome: a real-world, retrospective, cohort study. eClinicalMedicine. July, 4, 2023. DOI: https://doi.org/10.1016/j.eclinm.2023.102062
5	ClinicalTrials.gov Identifier: NCT04968912 Available at: https://clinicaltrials.gov/study/NCT04968912. Last accessed: March 2025.
6	Nat Rev Rheumatol 20, 158–169 (2024). https://doi.org/10.1038/s41584-023-01057-6
7	Beydon, M., McCoy, S., Nguyen, Y. et al. Epidemiology of Sjögren syndrome.
8	Carsons SE, Patel BC. Sjogren Syndrome. [Updated 2023 Jul 31]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK431049/
9	Hackett KL, et al. Arthritis Care Res (Hoboken). 2012;64(11):1760-1764.
10	ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: March 2025
11	ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: March 2025
12	ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114. Last accessed: March 2025
13	ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050. Last accessed: March 2025
14	ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634 Last accessed: March 2025.
15	ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517. Last accessed: March 2025
16	ClinicalTrials.gov Identifier: NCT06028438. Available at: https://clinicaltrials.gov/study/NCT06028438. Last accessed: March 2025
17	ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: March 2025.
18	Lobato G, Soncini CS. Relationship between obstetric history and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x.
19	Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499.

Media contact:
Bridget Kimmel
bkimmel@its.jnj.com

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

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FAQ

What are the key Phase 2 DAHLIAS study results for JNJ's nipocalimab in Sjögren's disease?

The study showed >70% improvement in disease activity at Week 24 vs placebo, >77% IgG reductions, and positive trends across secondary endpoints, with consistent safety profile.

What regulatory designations has JNJ's nipocalimab received for Sjögren's disease treatment?

Nipocalimab has received both FDA Fast Track designation and Breakthrough Therapy designation, becoming the first and only treatment to receive both for Sjögren's disease.

What is the current status of JNJ's nipocalimab clinical trials for Sjögren's disease?

JNJ is actively enrolling patients in the Phase 3 DAFFODIL study, following successful Phase 2 DAHLIAS results.

What unmet medical need does JNJ's nipocalimab address in Sjögren's disease?

Nipocalimab addresses the lack of FDA-approved advanced treatments for Sjögren's disease, which can cause serious complications including 20x higher risk of B-cell lymphomas.