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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

The pairing of two Bristol Myers Squibb immunotherapies is now permitted for the first-line treatment of two types of gastrointestinal cancers, after regulatory decisions handed out this past week that convert the accelerated approval status of the drug combination to full FDA approval in both indications. The drugs, Opdivo and Yervoy, belong to the class of medicines called checkpoint inhibitors. On April 8, the FDA approved use of this drug combination for adults and children age 12 and older whose colorectal cancer has metastasized or cannot be removed by surgery. The cancer must also be microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), references to biomarkers that indicate the stability of DNA in a tumor. The regulatory submission was based on the results of an open-label Phase 3 test that compared the Opdivo/Yervoy combination to either Opdivo alone or chemotherapy. Results showed the immunotherapy combination led to a 38% reduction in the risk of disease progression or death compared to Opdivo monotherapy, and by 79% compared to chemo, both in the first-line setting. The study is ongoing to assess secondary measures, including overall survival. BMS said it will continue working with study investigators to present these data and longer-term follow-up results in the future. Sponsored Post Changes in Nurse Staffing Answer Clinician Demands The ongoing nursing shortage facilitates high turnover rates since nurses know they won’t have difficulties finding new jobs. In order to retain and attract staff, it’s in a facility’s best interest to understand what nurses want. By Tomasz Rezik On April 11, the FDA approved the combination of Opdivo and Yervoy for treating advanced cases of hepatocellular carcinoma. In the pivotal test in this indication, the drug combination led to median overall survival of 23.7 months. The comparator arm, in which patients received standard of care drugs sorafenib or levantinib, median overall survival was 20.6 months. At three years, the drug combination showed an overall survival rate of 38% versus 24% in the comparator arm. FDA approval of Opdivo plus Yervoy in hepatocellular carcinoma follows the European Commission’s early March approval of the drug combination in this indication. There’s plenty of news on the regulatory front. Here’s a recap of other recent highlights: New and Expanded Regulatory Approvals —Argenx drug Vyvgart Hytrulo received an additional approval for dosing of the product via a prefilled syringe. The drug, supplied in vials, previously received FDA approvals for the autoimmune conditions chronic inflammatory demyelinating polyneuropathy and generalized myasthenia gravis. Analysts say prefilled syringes bring patients a more convenient dosing option while giving Argenx the opportunity to get more market share by penetrating into earlier lines of therapy. Sponsored Post Understanding EGPA: The Role of Eosinophils and Advancements in Treatment Options FASENRA® (benralizumab) injection, for subcutaneous use, 30 mg is indicated for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). FASENRA provides a treatment option for HCPs to consider when managing this challenging disease. By FASENRA, Sponsored Content —Amgen’s Uplizna expanded its label to include the treatment of immunoglobulin G4-related disease (IgG4-RD), an immune-mediated inflammatory disorder that can affect multiple organs. The regulatory decision makes the intravenously infused antibody the first FDA-approved treatment for this rare and chronic disease. Uplizna was first approved in 2020 as a treatment for neuromyelitis optica spectrum disorder. —Bayer’s Viktrakvi now has full FDA approval for the treatment of NTRK gene fusion-positive solid tumors in adults and children without a known acquired resistance mutation. The drug received accelerated approval in this indication in 2018. —AstraZeneca and Daiichi Sankyo welcomed European Union approval of Datroway as a treatment for metastatic breast cancer that is HR positive and HER2 negative. Datroway received FDA approval in the same indication in January. The drug is an antibody drug conjugate designed to target the cancer protein TROP2. —Novartis drug atrasentan, brand name Vanrafia, was awarded accelerated approval as a new treatment for the kidney disorder immunoglobulin A nephropathy (IgAN). The drug is an oral small molecule designed to block the endothelin A receptor. It’s one of two IgAN nephropathy drugs that came via the $3.2 billion acquisition of Chinook Therapeutics in 2023. Novartis’s internal IgAN research yielded the complement inhibitor Fabhalta, which received accelerated approval in this indication last summer. —Speaking of Fabhalta, that Novartis drug expanded its label to include C3 glomerulopathy, making the twice-daily pill the first approved treatment for this rare kidney disorder. Fabhalta is a small molecule designed to block the complement protein C3. It was first approved in 2023 as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria. —Sanofi received FDA approval for fitusiran, brand name Qfitlia, as a treatment for both hemophilia A and B. The drug leverages RNA interference to lower levels of AT, a protein that inhibits blood clotting. The approval covers the treatment of adults and children age 12 and older with or without inhibitors, which are antibodies that can develop in hemophilia patients, making it more difficult to control bleeding. By contrast, new Pfizer drug Hympavzi is approved only for hemophilia A and B patients without inhibitors while Sanofi’s Alhemo is approved only for hemophilia A and B patients with inhibitors. —Blockbuster AstraZeneca cancer immunotherapy Imfinzi landed European Union approval for the treatment of limited-stage small cell lung cancer. This new approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. The FDA approved Imfinzi for this indication last December. —Imfinzi also added perioperative treatment of muscle invasive bladder cancer to its list of FDA-approved indications. The newest FDA nod covers use of the drug in combination with chemotherapy before surgery to remove the bladder, followed by Imfinzi as an adjuvant monotherapy. In the pivotal study supporting this new indication, results showed a 32% reduction in the risk of cancer recurrence and a 25% reduction in the risk of death compared to neoadjuvant chemotherapy alone. —The European Commission approved Pfizer vaccine Abrysvo for protection against respiratory syncytial virus (RSV). The regulatory decision expands the vaccine’s approval to adults ages 18 to 59. European regulators initially approved the vaccine in 2023 for preventing lower respiratory tract disease in adults age 60 and older. Abrysvo won FDA approval the same year. —The FDA expanded approval of Novartis’s Pluvicto to include use as an earlier line of treatment — after anti-androgen drugs and before chemotherapy — for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Pluvicto was initially approved in 2022 for PSMA-positive mCRPC that has already been treated with an anti-androgen drug and chemotherapy. Novartis said this expansion is important because about half of patients do not live long enough to receive a second-line treatment. —Exelixis cancer drug Cabometyx expanded its FDA-approved uses to advanced cases of pancreatic neuroendocrine tumors (pNET) and extra-pancreatic neuroendocrine tumors (epNET). The approval covers adults and children age 12 and older. Cabometyx is a small molecule designed to block enzymes that support cancer growth. The drug was first approved in 2016 for advanced renal cell carcinoma. —GSK landed FDA approval for Blujepa, an oral antibiotic developed to treat uncomplicated urinary tract infections. While drugs are already available for these infections, they can become resistant to treatment. Blujepa is designed to block two enzyme targets key to bacterial DNA replication, an approach intended to lower the potential for drug resistance. —The European Commission approved Capvaxive, a pneumococcal vaccine developed by Merck to protect against the 21 Streptococcus pneumoniae serotypes that lead to the majority of cases of invasive pneumococcal disease including eight that are not covered by any other available pneumococcal vaccine. The European regulatory decision covers adults age 18 and older. Capvaxive received FDA approval last July. —Alynlam Pharmaceuticals drug Amvuttra received FDA approval for treating cardiomyopathy caused by transthyretin amyloidosis (ATTR). The RNA interference drug was first approved in 2022 for the treatment of polyneuropathy caused by ATTR. In the cardiomyopathy indication, it will compete with Pfizer’s Vyndaqel and Vyndamax, which are established as the standard of care, as well as Attruby, a BridgeBio Pharma drug that received FDA approval last fall. —Bristol Myers Squibb’s Breyanzi received European Commission approval for relapsed or refractory follicular lymphoma. The CAR T-therapy received accelerated FDA approval in this indication last May. —Geron Corporation’s Rytelo received European Commission approval for the treatment of transfusion-dependent anemia caused by lower-risk myelodysplastic syndrome, a progressive type of blood cancer. The intravenously infused drug is the first telomerase inhibitor to reach the market. Rytelo landed FDA approval last June. —Neffy, an epinephrine nasal spray that ARS Pharmaceuticals developed to treat allergic reactions to insect bites, medications, and food, expanded its FDA approval to include patients weighing 15 to 30 kilograms (about 33 to 60 pounds). The product’s initial approval last year covered its use in patients weighing 30 kg or more. —Roche subsidiary Genentech received FDA approval for tenecteplase, brand name TNKase, as a treatment for acute ischemic stroke in adults. Genentech said TNKase is the first stroke medicine approved by the FDA in nearly 30 years. It’s the second approved indication for the clot-busting drug. The FDA approved it in 2000 for treating acute myocardial infarction. —A Neurotech Pharmaceuticals cell and gene therapy administered via a surgical implant received FDA approval for the treatment of macular telangectasia type 2. The therapy, brand name Encelto, is the first approved treatment for this rare vision-loss disorder. Regulatory Setbacks —The FDA missed the April 1 regulatory decision target date for Novavax’s Covid-19 vaccine. Politico reported the holdup was due to the intervention of Sara Brenner, the FDA’s principal deputy commissioner, who asked for more data. Novavax said it has responded to all of the FDA’s information requests and believes the application is ready for approval based on the submitted Phase 3 data. Novavax’s protein-based vaccine initially received FDA emergency use authorization in 2022, a status that it retained under amended authorizations in 2023 and last year, both times for new formulas that covered the prevalent variants at the time. Full FDA approval would put the vaccine on par with the approved messenger RNA vaccines marketed by Pfizer and Moderna. Novavax’s Covid-19 vaccine will be co-commercialized with Sanofi under a partnership announced last year. —Roche paused European clinical tests of the Duchenne muscular dystrophy gene therapy Elevidys, the pharmaceutical giant said in a letter to the World Duchenne Organization. The move follows Sarepta Therapeutics’ recent report of a liver failure-associated patient death. Roche said the European Medicines Agency asked for a clinical hold on four studies until the inquiry into the cause of death is complete. Under a 2019 deal with Sarepta, Roche holds rights to Elevidys outside of the U.S. —For the second time, the FDA has rejected Aldeyra Therapeutics drug reproxalap as a treatment for dry eye disease. The agency turned down an application for the drug in 2023. According to Aldeyra, the FDA’s latest complete response letter said the new drug application failed to demonstrate efficacy. The regulator asked the company to run at least one more clinical trial. Among the concerns raised by the FDA is differences in baseline scores, which may have affected interpretation of trial results. Aldeyra said results from two ongoing studies are expected later in the current quarter, potentially paving the way for a mid-year resubmission of the application. —The European Medicines Agency refused marketing authorization for Eli Lilly Alzheimer’s disease drug Kisunla. Safety was the main reason cited, particularly the brain inflammation and bleeding that is a known complication risk associated with all drugs in the same class of Alzheimer’s medicines. The agency said the drug’s benefits were not enough to outweigh the potentially fatal safety risks. The FDA approved Kisunla last July. —In other Alzheimer’s drug news, Eisai again failed to persuade Australia’s drugs regulator to register Leqembi. The pharma company had asked the Therapeutics Goods Administration (TGA) to reconsider its October 2024 decision to not register the drug in Australia. Eisai’s request for reconsideration covered a narrower indication, but Eisai said the TGA did not agree that safety has been established for these patients.