Warm autoimmune hemolytic anemia

With UPLIZNA receiving a label expansion that opens the door to a previously untapped rare disease market, Amgen is seeing strong returns on its USD 27.8 billion acquisition of Horizon Therapeutics. The drug has become the first to receive FDA approval specifically for treating immunoglobulin G4-related disease (IgG4-RD). LAS VEGAS, April 14, 2025 /PRNewswire/ -- IgG4-related disease (IgG4-RD) is a systemic fibroinflammatory condition marked by the accumulation of IgG4-positive plasma cells in affected tissues, sometimes accompanied by elevated blood levels of IgG4. The disease can impact nearly any organ, with common sites including the pancreas, bile ducts, salivary and lacrimal glands, orbits, kidneys, lungs, and the retroperitoneal space. Due to its relatively recent identification, lack of widespread clinical awareness, and often subtle or slow-developing symptoms, IgG4-RD is likely underdiagnosed and underreported. It occurs more frequently in men, with most diagnoses in adults between their 30s and 50s, though cases have also been documented in children. In 2024, there were ~ 149K diagnosed prevalent cases of IgG4-RD across the 7MM, with the United States representing the largest share of these cases. In patients with IgG4-related disease, treatment may not always be necessary. In cases where individuals are asymptomatic, a watchful waiting approach can be appropriate. However, when vital organs are involved or symptoms are present, timely treatment becomes crucial, as IgG4-RD can lead to significant organ damage and dysfunction. Glucocorticoids are considered the first-line treatment, as outlined in the International Consensus Statement on IgG4-RD management. They typically produce rapid and substantial clinical improvement in both pancreatic and extra-pancreatic manifestations. To help maintain remission and reduce long-term steroid use, various glucocorticoid-sparing agents—such as azathioprine, mycophenolate mofetil, methotrexate, cyclophosphamide, and bortezomib—have been utilized across different organ systems, though their effectiveness varies. Steroid-sparing drugs like azathioprine, mycophenolate mofetil, and methotrexate are used to minimize the side effects of prolonged steroid therapy and support long-term disease control. However, the evidence supporting their efficacy remains limited. Rituximab has emerged as a promising newer option; it targets CD20-positive plasmablast precursors, leading to a reduction in plasmablasts and a subsequent decline in IgG4 production. To know more about IgG4-RD treatment options, visit @ New Treatment for IgG4-related Disease Recently, in April 2025, the FDA approved Amgen's anti-CD19 therapy, UPLIZNA, for the treatment of IgG4-related disease, making it the first and only approved treatment specifically for this condition, according to the company's announcement. This label expansion is supported by data from the Phase III MITIGATE trial, initially presented by Amgen in November 2024. The study showed that UPLIZNA reduced the risk of disease-related flares by 87% compared to placebo. Over the 52-week study period, only 7 out of 68 patients receiving UPLIZNA experienced flares, compared to 40 out of 67 in the placebo group—a difference Amgen highlighted as statistically significant. Additionally, 57.4% of patients treated with UPLIZNA achieved complete remission at the one-year mark versus just 22.4% of those on placebo. Nearly 90% of patients in the UPLIZNA arm were able to stop using glucocorticoids, compared to 37.3% in the placebo group. Within just 8 weeks of treatment, UPLIZNA recipients had a tenfold reduction in total glucocorticoid usage compared to placebo. Administered via intravenous infusion, UPLIZNA is a humanized monoclonal antibody that targets the CD19 protein found on specific immune cells. By binding to CD19, it depletes both immature and mature B cells, thereby addressing a key disease mechanism in IgG4-RD, which involves the widespread infiltration of B cells into affected tissues, leading to organ damage. Amgen recorded USD 379 million in UPLIZNA sales in 2024 from its original indication alone. However, the recent approval for IgG4-related disease presents a significant growth opportunity, as this new market includes a treatable patient population that is roughly twice as large as that for NMOSD. Learn more about the FDA-approved IgG4-RD drugs @ Drugs for IgG4-related Disease Treatment Apart from Amgen, key players such as Zenas Biopharma, Bristol Myers Squibb and Sanofi are also evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products to treat IgG4-RD. The treatment pipeline for IgG4-RD includes several promising therapies in advanced stages of development, such as ZB012 (obexelimab) and Rilzabrutinib, among others. These emerging treatments hold the potential to significantly improve patient outcomes and address existing gaps in care. Discover which therapies are expected to grab major IgG4-RD market share @ IgG4-related Disease Market Report Obexelimab (XmAb5871) is a dual-function monoclonal antibody that targets both CD19 and FcγRIIb—proteins found widely on B cells—aiming to suppress B-cell activity involved in various autoimmune disorders without destroying the cells. Its distinctive mechanism and subcutaneous self-injection format position it as a promising therapy to effectively address the role of B cells in chronic autoimmune conditions. Zenas is currently advancing obexelimab through multiple Phase II and III clinical trials for a range of autoimmune diseases, including IgG4-related disease, multiple sclerosis, systemic lupus erythematosus, and warm autoimmune hemolytic anemia. In August 2023, The Lancet Rheumatology published Phase II trial results showing its potential in treating IgG4-RD. These findings have led to an ongoing Phase III trial focused on evaluating the safety and efficacy of obexelimab via subcutaneous injection in patients with IgG4-RD. Rilzabrutinib (also known as PRN1008) is a selective, orally available, reversible covalent inhibitor of Bruton's Tyrosine Kinase (BTK), exhibiting a potent IC50 of 1.3 nM. It targets a cysteine residue on BTK, resulting in a slow dissociation rate—about 79% of BTK binding remains in peripheral blood mononuclear cells (PBMCs) 18 hours after the compound is washed out in vitro. This covalent binding is fully reversible upon denaturation of the protein. Functionally, rilzabrutinib suppresses anti-IgM-induced human B-cell proliferation and inhibits CD69 expression on B cells, with IC50 values of 5 ± 2.4 nM and 123 ± 38 nM, respectively, under 10% serum conditions. In August 2020, Sanofi entered into a definitive agreement to acquire Principia Biopharma, a deal approved unanimously by both companies' boards. The acquisition strengthened Sanofi's pipeline in immunology by integrating Principia's BTK inhibitors—including rilzabrutinib—into its broader strategy to develop next-generation therapies for autoimmune diseases. Discover more about drugs for IgG4-RD in development @ IgG4-related Disease Clinical Trials The anticipated launch of these emerging therapies for IgG4-RD are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the IgG4-RD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for IgG4-RD is expected to grow from USD 170 million in 2024, with a significant CAGR by 2034. This growth can be attributed to the approval of new therapies with improved safety and tolerability, which will significantly impact the treatment landscape in the coming years. DelveInsight's latest published market report, titled as IgG4-related Disease Market Insight, Epidemiology, and Market Forecast – 2034 , will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the IgG4-RD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The IgG4-RD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of IgG4-RD Gender-specific Diagnosed Prevalent Cases of IgG4-RD Age-specific Diagnosed Prevalent Cases of IgG4-RD The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM IgG4-RD market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this IgG4-RD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the IgG4-RD market. Also, stay abreast of the mitigating factors to improve your market position in the IgG4-RD therapeutic space. Related Reports IgG4-related Disease Epidemiology IgG4-related Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted IgG4-RD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan. IgG4-related Disease Pipeline IgG4-related Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key IgG4-RD companies, including Amgen, Sanofi, Zenas Biopharma, Bristol-Myers Squibb, among others. Warm Autoimmune Hemolytic Anemia Market Warm Autoimmune Hemolytic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key wAIHA companies including ZENAS BIOPHARMA, JOHNSON & JOHNSON INNOVATIVE MEDICINE, NOVARTIS, MORPHOSYS, SANOFI, ALPINE IMMUNE SCIENCES , among others. Neuromyelitis Optica Market Neuromyelitis Optica Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key neuromyelitis optica companies including Alexion Pharmaceutical, AstraZeneca , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

New compelling results demonstrate 18 months of both sustained reduction in immunoglobulin G antibodies and sustained improvement in gMG symptoms in pivotal Vivacity-MG3 study and open-label extension phase Up to 128 weeks and 180 patient years of follow-up in the open-label extensiona confirm a safety profile consistent with the Phase 3 Vivacity-MG3 study 45% of the patients receiving steroids at open-label extension baseline were able to decrease or discontinue their steroid use Additionally, the nipocalimab plus standard of care (SOC) group demonstrated four times greater odds of improving and maintaining the strength and function of different muscle groups as measured by QMGb response versus placebo plus SOC in the 24-week double blind phase of the study SPRING HOUSE, Pa., April 8, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced results from additional analyses of the Phase 3 Vivacity-MG3 double-blind study and the ongoing open-label extensiona (OLE), evaluating the long-term efficacy and safety of investigational nipocalimab in a broad population of antibody-positive (anti-AChR+, anti-MuSK+, anti-LRP4+) adults with generalized myasthenia gravis (gMG).1,2 Patients treated with nipocalimab plus standard of care (SOC) maintained improvements in their MG-ADLc and QMGb scores over 84 weeks with sustained reductions in total immunoglobulin G (IgG).1 These data are included in a presentation (Session 7 #022) and are among 12 abstracts that Johnson & Johnson will present at the American Academy of Neurology (AAN) 2025 Meeting in San Diego, California, which includes an oral presentation on QMG score improvements from the double-blind phase of the Phase 3 Vivacity-MG3 study. "The sustained disease control seen over 84 weeks for nipocalimab is a key result given the chronic course of generalized MG and the significant burden on people living with this condition," said Constantine Farmakidis M.D., Associate Professor of Neurology at University of Kansas Medical Centerd. "Overall, I am encouraged by these results that show improvement in disease control as measured by the MG-ADL and QMG scores across a broad population seropositive for AChR, MuSK, or LRP4 autoantibodies." Nipocalimab demonstrated a mean change in MG-ADL of -5.64 (p<0.001) from the double-blind baseline after 60 weeks in the OLE for study participants receiving nipocalimab and SOC, and -6.01 (p<0.001) mean change for study participants who transitioned from placebo and SOC to nipocalimab and SOC.1 In the antibody-positive population, 45% of patients receiving steroids at the OLE baseline were able to decrease or discontinue steroids at the time of this data cut by more than half of the baseline dose.1 Among these patients, the mean dose of prednisone decreased from 23 to 10 mg per day.1 Nipocalimab had a consistent and tolerable safety profile throughout the OLE phase.1 Additional findings from the Phase 3 Vivacity-MG3 double-blind study indicate that patients treated with nipocalimab plus SOC achieved statistically significant improvements in their QMG score by -4.9 versus placebo plus SOC (p<0.001) over weeks 22 and 24.2 Patients in the nipocalimab plus SOC treatment group were four times more likely to sustain symptom improvement at 20 weeks compared to the placebo plus SOC group, as measured by a three or greater point improvement on the QMG score.2 Results show significantly more patients treated with nipocalimab (36.4%,) versus placebo (10.5%, p<0.001) spent greater than 75% of study duration demonstrating improvements in the QMG score.2 A reduction of more than three points in the QMG score indicates a decrease in the severity of the patient's symptoms as a result of improvements in muscle strength, allowing patients to carry out important daily activities such as swallowing and chewing.3,4 "People living with generalized MG around the world endure daily challenges, such as difficulties swallowing, impaired speech and muscle weakness. They deserve additional, effective treatment options that help address these challenges and provide sustained disease control and stability over time," said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson Innovative Medicine. "These positive data underscore our commitment to helping develop potential innovative therapeutic options for patients living with autoantibody diseases, including gMG." Editor's notes: 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.5,6,7 The disease impacts an estimated 700,000 people worldwide.5 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently starts in young women and older men.8 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential.9,10,11 Approximately 10 to 15% of new cases of MG are diagnosed in adolescents (12 – 17 years of age).12,13,14 Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the U.S. diagnosed in girls.15,16,17 Initial disease manifestations are usually ocular, but 85 percent of MG patients experience additional advancements to the disease manifestations – referred to as generalized myasthenia gravis (gMG). This is characterized by severe muscle weakness of the skeletal muscles and difficulties in speech and swallowing.18,19,20,21,22 Approximately 100,000 individuals in the U.S. are living with gMG.23 Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.24 Currently, SOC treatments for adolescents with gMG are extrapolated from adult trials.14 Other than symptomatic treatments, there are no approved FcRn blockers for adolescents with gMG in the United States.14 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 SOC therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.25,26 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.25 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo).25 The primary endpoint of the study was mean change in MG-ADLb 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.26 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.26,27,28,29,30,31,32,33,34 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.35,36 The U.S. FDA and 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 or at 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 , 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., Long-Term Safety and Efficacy of Nipocalimab in Generalized Myasthenia Gravis: Vivacity-MG3 Open-Label Extension Phase Results. Abstract #022 for poster presentation at 2025 American Academy of Neurology Congress. April 2025. 2 Nowak R et al., Efficacy of Nipocalimab, a Novel Neonatal Fragment Crystallizable Receptor Blocker, as Measured using Quantitative Myasthenia Gravis Assessment: Findings from the Phase 3 Placebo-Controlled Vivacity-MG3 Study. Abstract #001 for oral presentation at 2025 American Academy of Neurology Congress. April 2025. 3 Wolfe GI Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. 4 Yin J, et al. A multicenter, randomized, open-label, phase 2 clinical study of telitacicept in adult patients with generalized myasthenia gravis. Eur J Neurol. 2024 Aug;31(8):e16322. doi: 10.1111/ene.16322. 5 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. 2020;5(100063). . 6 Bacci ED et al. Understanding side effects of therapy for myasthenia gravis and their impact on daily life. BMC Neurol. 2019;19(1):335. 7 Wiendl, H., et al., Guideline for the management of myasthenic syndromes. Therapeutic advances in neurological disorders, 16, 17562864231213240. . Last accessed: April 2025. 8 Bubuioc A, et al. The epidemiology of myasthenia gravis. Journal of Medicine & Life (2021). Jan-Mar;14(1):7-16. doi: 10.25122/jml-2020-0145. 9 Ye, Yun et al. Epidemiology of myasthenia gravis in the United States. Frontiers in neurology vol. 15 1339167. 16 Feb. 2024, doi:10.3389/fneur.2024.1339167. 10 Dresser, Laura et al. Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. Journal of clinical medicine vol. 10,11 2235. 21 May. 2021, doi:10.3390/jcm10112235. 11 J&J. Data on file. 12 Evoli A, Batocchi AP, Bartoccioni E, Lino MM, Minisci C, Tonali P. Juvenile myasthenia gravis with prepubertal onset. Neuromuscul Disord. 1998 Dec;8(8):561-7. doi: 10.1016/s0960-8966(98)00077-7. 13 Evoli A. Acquired myasthenia gravis in childhood. Curr Opin Neurol. 2010 Oct;23(5):536-40. doi: 10.1097/WCO.0b013e32833c32af. 14 Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101. 15 Haliloglu G, Anlar B, Aysun S, Topcu M, Topaloglu H, Turanli G, Yalnizoglu D. Gender prevalence in childhood multiple sclerosis and myasthenia gravis. J Child Neurol. 2002 May;17(5):390-2. doi: 10.1177/088307380201700516. 16 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. 17 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. 18 National Institute of Neurological Disorders and Stoke. Myasthenia Gravis. Available at: . Last accessed: April 2025. 19 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. . 20 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. PMID: 12580710. 21 Myasthenia gravis fact sheet. Retrieved April 2024 from . 22 Myasthenia Gravis: Treatment & Symptoms. (2021, April 7). Retrieved April 2024 from . 23 DRG EPI (2021) & Optum Claims Analysis Jan 2012-December 2020. 24 O'Connell K, Ramdas S, Palace J. Management of Juvenile Myasthenia Gravis. Front Neurol. 2020 Jul 24;11:743. doi: 10.3389/fneur.2020.00743. PMID: 32793107; PMCID: PMC7393473. 25 Antozzi, C et al., Nipocalimab in Generalized Myasthenia Gravis: Results from a Double-Blind, Placebo-Controlled, Randomized Phase 3 Study. Abstract at 2024 European Academy of Neurology Congress. June 2024. 26 ClinicalTrials.gov Identifier: NCT04951622. Available at: . Last accessed: April 2025. 27 ClinicalTrials.gov. NCT03842189. Available at: . Last accessed: April 2025. 28 ClinicalTrials.gov Identifier: NCT05327114. Available at: . Last accessed: April 2025. 29 ClinicalTrials.gov Identifier: NCT04119050. Available at: . Last accessed: April 2025. 30 ClinicalTrials.gov Identifier: NCT05379634. Available at: . Last accessed: April 2025. 31 ClinicalTrials.gov Identifier: NCT05912517. Available at: . Last accessed: April 2025. 32 ClinicalTrials.gov Identifier: NCT06028438. Available at: . Last accessed: April 2025. 33 ClinicalTrials.gov Identifier: NCT04968912. Available at: . Last accessed: April 2025. 34 ClinicalTrials.gov Identifier: NCT04882878. Available at: . Last accessed: April 2025. 35 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. 36 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. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The therapy leverages Sanofi’s tailored covalency technology. Credit: rafaelnlins/Shutterstock. Sanofi’s oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor rilzabrutinib has gained US Food and Drug Administration (FDA) orphan drug designation (ODD) to treat two rare conditions: IgG4-related disease (IgG4-RD) and warm autoimmune haemolytic anaemia (wAIHA). IgG4-RD is a chronic fibro-inflammatory condition that can result in organ damage and sometimes fatal outcomes, and wAIHA is a life-threatening autoimmune disorder characterised by the premature destruction of red blood cells. Outcomes from a Phase IIb trial in wAIHA patients demonstrated meaningful data on disease markers and response rate. A Phase IIa trial in patients with IgG4-RD demonstrated that the therapy for 52 weeks decreased disease flare-ups, disease markers and the need for glucocorticoids. The safety profile observed in both trials was consistent with previous trials. The therapy is also being reviewed in the European Union (EU), China and the US for immune thrombocytopenia (ITP). The FDA has set a target action date of 29 August 2025 for its decision on ITP, having previously granted the drug fast-track status. The therapy has also gained ODD status for ITP treatment in the EU, Japan and the US. Sanofi rare diseases development global head Karin Knobe stated: “Orphan drug designation for these two rare, immune-mediated conditions validates our ongoing commitment to pursuing potential first and best-in-class medicines for diseases that affect small populations but persist with unmet medical need. “Our continued exploration of rilzabrutinib across multiple indications speaks to our belief in its potential for multi-immune modulation, as well as our belief in supporting treatment options, no matter how rare a condition.” The company’s tailored covalency technology, which was applied to rilzabrutinib, allows for selective blocking of the BTK target and may decrease the off-target side effect risk. The latest development follows Sanofi’s recent FDA approval for its haemophilia treatment , Qfitlia (fitusiran).