RegulationPriority Review (United States), Breakthrough Therapy (United States), Orphan Drug (United States), PRIME (European Union), Priority Review (China), Conditional marketing approval (China), Orphan Drug (Japan), Fast Track (United States)

Structure/Sequence

Sequence Code 25650568H

Source: *****

Sequence Code 25650570L

Source: *****

Clinical Trials associated with Emapalumab-LZSG

NCT06996119

/ Not yet recruitingPhase 1IIT

Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation

This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after reduced-intensity donor (allogeneic) hematopoietic cell transplant (HCT). Giving chemotherapy, such as fludarabine, melphalan, or busulfan, before a donor [peripheral blood stem cell] transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When healthy stem cells for a donor are infused into a patient (allogeneic HCT), they may help the patient's bone marrow make more healthy cells and platelets. Allogeneic HCT is an established treatment, however, GVHD continues to be a major problem of allogeneic HCT that can complicate therapy. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Emapalumab binds to an immune system protein called interferon gamma. This may help lower the body's immune response and reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Mycophenolate mofetil is a drug used to prevent GVHD after organ transplants. It is also being studied in the prevention of GVHD after stem cell transplants for cancer, and in the treatment of some autoimmune disorders. Mycophenolate mofetil is a type of immunosuppressive agent. Giving emapalumab with post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil may be safe, tolerable and/or effective in preventing GVHD in patients with AML or MDS after a reduced-intensity allogeneic HCT.

Start Date25 May 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT07244419

/ RecruitingEarly Phase 1IIT

Emapalumab for the Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients

The investigators hypothesize that graft rejection after hematopoietic stem cell transplant (HSCT) is primarily driven by interferon gamma, and prophylactic interferon gamma inhibition in high-risk patients will prevent graft rejection. Additionally, knowledge of emapalumab PK/PD and in vitro mechanistic effects of emapalumab in this novel setting will guide optimization of dosing regimens and treatment approaches in future studies.

Start Date07 Jan 2026

Sponsor / Collaborator

Children's Hospital & Medical Center

[+1]

NCT07202598

/ RecruitingPhase 2IIT

A Phase 2 Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis

Background:
Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), also known as Autoimmune polyendocrine syndrome type-1 (APS-1), is a disease that causes the immune system to attack parts of a person s body. In some people, APECED attacks the small intestine; this causes an illness called enteritis.
Objective:
To test a drug (emapalumab) in people with enteritis caused by APECED.
Eligibility:
People aged 2 to 75 years with APECED and enteritis. They must also be enrolled in protocol 11-I-0187.
Design:
Participants will have 10-13 study visits in an 18-month period.
Participants will be screened. They will have a physical exam with blood tests. These tests will be repeated at every study visit. They will have a test of their heart function. This will be at screening and prior to drug administration.
Other tests are optional: Participants may have imaging exams and a test of lung function. They may have an endoscopy, which is an exam of their digestive tract. Participants may provide samples of urine, stool, nail clippings, saliva, vaginal fluid, or skin. Photos may be taken of their skin or scalp. These tests may be repeated at some visits.
Emapalumab is given through a tube attached to a needle inserted into a vein. All participants will receive 7 doses: 2 on their first study visit; then 1 each at 30-day intervals. Some participants will have an observation period before they begin taking the drug; in those situations, they will either be seen in person or via video visit every 2 months before starting emapalumab to see how their symptoms change over time.
Participants will have a follow-up visit 1 month after their last dose. Then they will have 2 telehealth visits at 30-day intervals. They will have a final clinic visit 1 year after their first dose.
...

Start Date12 Nov 2025

Sponsor / Collaborator

National Institute of Allergy & Infectious Diseases

100 Clinical Results associated with Emapalumab-LZSG

100 Translational Medicine associated with Emapalumab-LZSG

100 Patents (Medical) associated with Emapalumab-LZSG

Literatures (Medical) associated with Emapalumab-LZSG

10 Mar 2026·Blood Advances

Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study

Article

Author: Isakoff, Michael ; Carter, John ; Leiding, Jennifer W. ; Ray, Anish K. ; Intzes, Stefanos ; Tandra, Anand ; Oladapo, Abiola ; Hermiston, Michelle L. ; Gidvani-Diaz, Vinod K ; Hays, Allyson ; Rothman, Jennifer A. ; Cooper, Robert ; Sarangi, Susmita ; Walkovich, Kelly J. ; Satwani, Prakash ; Ward, Brant ; Chandrakasan, Shanmuganathan ; Henry, Michael ; Panigrahi, Arun ; Jordan, Michael B. ; Isakoff, Michael S. ; Yee, John D. ; Draper, Lauren ; Bhatt, Sima ; Bhatla, Deepika ; Gloude, Nicholas ; Hinson, Ashley P. ; Saldaña, Blachy Dávila ; Pednekar, Priti ; Weinstein, Joanna ; Modica, Renee ; Patel, Sachit A. ; Behrens, Edward M. ; Eckstein, Olive S. ; Hanna, Rabi ; Nakano, Taizo A. ; Chien, May ; Gulati, Nitya ; Allen, Carl E. ; Riskalla, Mona ; Haines, Hilary ; Zoref-Lorenz, Adi ; Baker, Ashley

Abstract:

Malignancy-associated hemophagocytic lymphohistiocytosis (mHLH), a hyperinflammatory syndrome, has poor prognosis and no standard therapy. Emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon gamma, is approved for treating primary hemophagocytic lymphohistiocytosis (pHLH). REAL-HLH, a retrospective chart review conducted across 33 US hospitals, evaluated real-world treatment patterns and outcomes in patients treated with ≥1 dose of emapalumab between 20 November 2018 and 31 October 2021. Data are presented for the subset of patients with mHLH. Overall, 51 of 105 (48.6%) patients were categorized as having secondary HLH, of which 17 of 51 patients had HLH associated with an underlying malignancy (mHLH). At HLH diagnosis, the median age (range) was 15.0 (3.0-27.0) years, 6 of 14 (42.9%) patients with available data had a positive Optimized HLH Inflammatory index, indicating pathologic inflammation; 9 of 17 (52.9%) had infections; and 10 of 17 (58.8%) received emapalumab in an intensive care unit. Emapalumab was primarily initiated for treating refractory (10/17; 58.8%) or progressive (3/17, 17.7%) disease. Most patients received HLH-related therapies before (16/17; 94.1%) and/or concurrent with (15/17; 88.2%) emapalumab. Treatment with emapalumab-containing regimens normalized most key laboratory parameters and maintained normal values for others (fibrinogen [11/13; 84.6%], absolute neutrophil count [6/10; 60%], and chemokine ligand 9 [7/8; 87.5%]) per physician assessment. Overall survival at the end of follow-up and 12-month survival probability from emapalumab initiation were 23.5% and 22.1%, respectively. In conclusion, emapalumab-containing regimens normalized, improved, or maintained normal values for most laboratory parameters in patients with mHLH. Future studies are warranted to establish appropriate emapalumab dosing and utility in this high-risk population.

01 Feb 2026·Transplantation and Cellular Therapy

Management Practices of CAR T-cell-Related Inflammatory Toxicities: A Survey of Pediatric CAR T-cell Providers

Article

Author: Ombrello, Amanda K ; Chaudhury, Sonali ; McNerney, Kevin O ; Diorio, Caroline ; Graham, Andrea K ; Maude, Shannon L ; Talleur, Aimee C ; Shah, Nirali N ; Gardner, Rebecca A ; Sanchez-Pinto, Lazaro N ; Annesley, Colleen ; Schultz, Liora M

Immune related adverse events (IRAE) are the most common nonhematologic adverse effects of chimeric antigen receptor (CAR) T-cell therapies. These events can be severe and refractory to upfront management. Tocilizumab is currently the only FDA-approved drug for the treatment of CAR T-cell induced inflammatory toxicities. However, an increasing number of anti-inflammatory therapies are now utilized for prevention and treatment of severe or refractory IRAE. Emerging evidence suggests the potential benefit of prophylactic or preemptive use of anti-inflammatory agents to mitigate severe toxicities. Guidance on the use of alternative anti-inflammatory therapies, preventative strategies, and management of refractory CAR T-cell toxicities is limited, with varying recommendations in society guidelines and the FDA labels for tisagenlecleucel and tocilizumab. Furthermore, there is a lack of pediatric-specific guidance. The primary objective of this study was to characterize CD19-CAR T-cell toxicity management practices for children and young adults with B-acute lymphoblastic leukemia by pediatric CAR T prescribers. A survey consisting of 28 questions focused on CAR T-cell toxicity management practices was distributed to pediatric CAR T-cell treatment centers with responses received between February and March 2024. Sixty unique responses with adequate data were received from 46 institutions. Forty-four respondents (73%) indicated using a preemptive treatment approach, defined as immunomodulatory therapy initiated for low grade toxicity to prevent the development of worsening toxicity, of which 33 (75%) used for high-risk patients and 9 (21%) used for all patients. Conversely, only 8 (14%) respondents indicated using a prophylactic approach defined as immunomodulatory therapy initiated prior to the onset of inflammatory toxicities. The most common feature leading to a patient being considered high-risk was high disease burden. Fifteen percent of respondents initiated tocilizumab for cytokine release syndrome (CRS) at grade 1, 48% at grade 2, and 18.3% at grade 3. Steroids were initiated at grade 2 immune effector cell-associated neurotoxicity syndrome (ICANS) in 43% of respondents and grade 3 in 28% of respondents. Most respondents had experience using alternative anti-inflammatory therapies, including anakinra (80%), siltuximab (50%), ruxolitinib (28%), emapalumab (22%), and dasatinib (15%). Anakinra was most often used for management of refractory CRS, refractory ICANS, or immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS). Most respondents indicated a need to prospectively study anakinra and emapalumab for treatment and/or prevention of CAR T inflammatory toxicities. Themes identified through analysis of semi-structured questions included the need for guidelines on use of alternative therapies, early preventative treatments, initial management of IRAE, and refractory IRAE management. This survey highlights significant practice variability in timing of initiation of CRS and ICANS treatment, frequent use of alternative anti-inflammatory therapies for CAR T toxicity management, and a consensus regarding the need for prospective study of therapies including anakinra and emapalumab. Survey results indicate a need for guidelines on management of CAR-induced inflammatory toxicities at their onset and in the refractory setting, and on the use of alternative therapies.

01 Feb 2026·JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

A clinically validated assay for rapid determination of type I and type II interferon activity in systemic inflammatory diseases

Article

Author: Yue, Jian ; Weng, Rachel ; Huang, Zhengping ; Wysocki, Christian ; Andzelm, Milena M ; Lam, Michael T ; Rimland, Casey A ; Ramanathan, Anusha ; Hsu, Evan ; Chou, Janet ; Platt, Craig D ; Chang, Joyce C ; Abraham, Roshini S ; Henderson, Lauren A ; Lee, Pui Y ; Basu, Amrita ; Alhezam, Musaab A ; Son, Mary Beth F ; Wobma, Holly ; Jiang, Connie L ; LeSon, Courtney ; Okada, Seigo ; Peng, Xiao P ; Brodeur, Kailey E

BACKGROUND:

Type I interferons (IFN-I) and type II interferon (IFN-γ) contribute to the pathogenesis of inflammatory diseases but measurement of these cytokines remains challenging in the clinical setting.

OBJECTIVE:

We aimed to develop a clinical test that rapidly captures the activity of IFN-I and IFN-γ.

METHODS:

We compared RNA sequencing data from healthy controls and patients with macrophage activation syndrome (MAS), multisystem inflammatory syndrome in children (MIS-C), and systemic lupus erythematosus (SLE) to evaluate biomarkers of IFN-I and IFN-γ signaling. We utilized a flow cytometry assay to measure IFN-inducible markers. The findings were verified by a clinical laboratory that independently developed and validated this assay.

RESULTS:

We identified CD274 (PD-L1) as a biomarker of IFN-γ signaling and confirmed CD169 (SIGLEC-1) as a readout of IFN-I activity. We utilized a flow cytometry assay to quantify CD169 and CD274 expression on monocytes and demonstrated the validity of this method for rapid screening of IFN dysregulation in patients with various inflammatory diseases including MAS, HLH, MIS-C, SLE, juvenile idiopathic arthritis, and juvenile dermatomyositis among others. We further demonstrated the utility of this test for assessment of IFN dysregulation in monogenic inflammatory diseases and for efficacy monitoring of medications that target the IFN pathways including Janus kinase inhibitors, emapalumab, and anifrolumab. Finally, we outlined the steps of assay validation and clinical implementation, and showed reproducible findings in a clinical laboratory.

CONCLUSION:

Flow cytometry analysis of CD169 and CD274 is an effective method to rapidly quantify IFN-I and IFN-γ activity in the clinical setting.

News (Medical) associated with Emapalumab-LZSG

18 Feb 2026

·www.prnewswire.com

Sobi Capital Markets Day 2026: Bringing brilliant ideas to life and building the next chapter with ambition to deliver SEK 55bn by 2030

STOCKHOLM, Feb. 18, 2026 /PRNewswire/ -- Sobi® (STO: SOBI) today hosts its Capital Markets Day, where senior management will provide an update on the company's strategy and outline its new mid-term outlook, aiming to achieve SEK 55bn revenue by 2030, while building a resilient platform for sustained growth beyond 2030. Sobi aims to have an adjusted EBITA margin in the upper 30s percentage of revenue by 2030. Sobi has delivered strong and profitable growth in recent years, transforming and diversifying its portfolio and positioning the company to deliver on six major launches. Additionally, Sobi is advancing priority development programs enabling longer term growth and continuing its international expansion. "Sobi today is a stronger, more global and more scientifically driven company," says Guido Oelkers, CEO of Sobi. "We have delivered on our commitments, built a diversified, robust foundation for future growth, and are entering the next phase of expansion with multiple launches and compelling opportunities to unlock growth well beyond 2030." Highlights of the Capital Markets Day Sobi shares its ambition to reach SEK 55bn in revenues by 2030, effectively doubling the company revenue driven by six major launches, continued strong performance of strategic products, and international expansion. Sobi outlines major launches either ongoing or in preparation, expected to drive the majority of growth to 2030, including Altuvoct®, Gamifant®, Aspaveli®, Tryngolza®, NASP, and pozdeutinurad, across haematology, immunology, and specialty care. Deep dives demonstrate Sobi's scientific leadership in: Uncontrolled and progressive gout and two potentially transformative treatment innovations in an area of high unmet patient needs. Severe hypertriglyceridemia, where acute pancreatitis is a major risk and a severe medical emergency, and ApoC-III inhibition with olezarsen has shown significant and meaningful reductions in acute pancreatitis risk. Precision medicine in sepsis: In biomarker-selected patients with interferon gamma-driven sepsis (the proof-of-concept EMBRACE study), Sobi will share topline data for empalumab demonstrating improvement in SOFA-based organ function (achieving primary endpoint at end of treatment in 60% patients receiving high dose emapalumab vs 40% in the standard of care plus placebo arm). High dose emapalumab additionally showed a 12-percentage point absolute mortality reduction after 28-days (40% mortality in the high dose emapalumab arm vs 52% in the standard of care plus placebo arm). Additional information provided New peak sales expectations for the following products: Haemophilia A franchise (Altuvoct and Elocta) greater than SEK 10bn, Aspaveli between SEK 7-10bn, Gamifant in HLH between SEK 5-7bn, NASP between SEK 4-6bn, Tryngolza greater than SEK 10bn, pozdeutinurad greater than SEK 10bn and Gamifant in IDS greater than SEK 10bn. R&D expenses to remain between 11-14 per cent of sales on a given year between now and 2030. Agenda in brief The Capital Markets Day includes presentations from Sobi's executive leadership on strategy, pipeline and innovation, commercial opportunities and financial outlook as well as disease deep dives with experts and leaders in their respective fields, and a concluding Q&A session. Pre-registration for online attendance: click here A recording of the presentations and supporting materials will be made available on . Sobi Sobi® is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2025 revenue amounted to SEK 28 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 08:00 CET on 18 February 2026. Gerard Tobin Head of Investor Relations This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB 21% more press release views with Request a Demo

Clinical Result

15 Jan 2026

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Hemophagocytic Lymphohistiocytosis Market Poised for Phenomenal Expansion in the US at a CAGR of 24.5% During the Forecast Period (2025-2034) | DelveInsight

The hemophagocytic lymphohistiocytosis market is expected to increase due to the launch of emerging therapies, such as plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), tadekinig alfa (r-hIL-18BP) (AB2 Bio), and others, as well as label expansions of current therapies. This is because of the higher patient burden, approval of newer therapies with high cost, and broader acceptance and availability of newly approved therapies. LAS VEGAS, Jan. 15, 2026 /PRNewswire/ -- DelveInsight's Hemophagocytic Lymphohistiocytosis Market Insights report includes a comprehensive understanding of current treatment practices, hemophagocytic lymphohistiocytosis emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophagocytic Lymphohistiocytosis Market Summary The market size for hemophagocytic lymphohistiocytosis was found to be USD 140 million in the United States in 2024. The HLH market remains highly unmet, with conventional therapies leading to high mortality (20–30% before HSCT) and only about 60% long-term survival due to toxicity, treatment failure, and relapse risk. Among the 7MM countries, there were 5,100 incident cases of HLH in the US in 2024. Leading hemophagocytic lymphohistiocytosis companies, such as TJ Biopharma, AB2 Bio, Electra Therapeutics, Novartis, and others, are developing new hemophagocytic lymphohistiocytosis treatment drugs that can be available in the hemophagocytic lymphohistiocytosis market in the coming years. The promising hemophagocytic lymphohistiocytosis therapies in clinical trials include Plonmarlimab (TJM2/TJ003234), Tadekinig alfa (r-hIL-18BP), ELA026, MAS 825, and others. Discover the new hemophagocytic lymphohistiocytosis treatment @ Key Factors Driving the Growth of the Hemophagocytic Lymphohistiocytosis Market Rising HLH Incidence: The increasing incidence of HLH, often triggered by underlying infections, autoimmune diseases, malignancies, or immuno-oncology treatments, has expanded the pool of patients needing diagnosis and therapy. Advances in Diagnostics and Early Detection: Improvements in diagnostic technologies, including genetic testing, immunologic profiling, and next-generation sequencing, enable earlier and more accurate detection of HLH. This allows timely intervention, which is critical given the rapid, life-threatening course of HLH. GAMIFANT Maintains First-in-Class Position in HLH: GAMIFANT (Sobi) remains the only approved therapy for primary HLH (since 2018) and has recently expanded its label, gaining US FDA approval in June 2025 for MAS associated with Still's disease. Following this approval, GAMIFANT has demonstrated stronger-than-projected market uptake, with double-digit growth in Constant Exchange Rates (CER). The company plans to pursue EU regulatory submission in 2026, indicating continued strategic expansion of its HLH and MAS portfolio. Launch of Emerging HLH Drugs: The dynamics of the HLH market are expected to change in the coming years due to the launch of emerging therapies such as plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), tadekinig alfa (r-hIL-18BP) (AB2 Bio), and others. Hemophagocytic Lymphohistiocytosis Market Analysis Management of HLH necessitates a comprehensive, multimodal strategy that integrates immunosuppressive, cytotoxic, and targeted therapies to control hyperinflammation, limit pathological immune activation, and restore immune homeostasis. Therapeutic decisions are guided by disease severity and the underlying trigger, with early, aggressive intervention essential to prevent irreversible organ damage. High-dose corticosteroids, particularly dexamethasone, constitute a foundational component of HLH treatment due to their potent anti-inflammatory effects and ability to suppress cytokine-mediated tissue injury. Etoposide-based regimens remain a cornerstone of first-line therapy, as they effectively eradicate cytotoxic lymphocytes and macrophages that are excessively activated and drive disease pathology. Immunosuppressive agents such as cyclosporine are commonly employed to suppress T-cell activation and reduce cytokine release, thereby dampening immune overactivation. Targeted biologic therapies, including rituximab for EBV-associated HLH and monoclonal antibodies such as alemtuzumab or antithymocyte globulin in refractory disease, enable more selective immune modulation. When infections serve as triggering factors, concurrent antimicrobial therapy, including antivirals and antibiotics, is critical to address both the precipitating cause and the downstream inflammatory cascade. Collectively, these treatment approaches aim to achieve disease stabilization and control, allowing eligible patients to proceed to definitive curative options such as hematopoietic stem cell transplantation (HSCT). GAMIFANT demonstrated strong commercial momentum in Q3 2025, generating USD 66.6 million in revenue, a 98% year-over-year increase. Revenue growth was driven by rising patient adoption, a favorable product mix, and the successful U.S. launch for macrophage activation syndrome (MAS) in Still's disease, making GAMIFANT the first approved therapy for both pediatric and adult patients. This milestone highlights GAMIFANT's broader applicability across the HLH–MAS disease spectrum and reinforces its long-term growth potential. Regulatory momentum remains positive, while active compassionate-use programs in Europe continue to reflect sustained clinician demand. Looking ahead, GAMIFANT revenues are expected to grow at a low double-digit CAGR at constant exchange rates, supported by global market expansion, improved patient access, and continued uptake across both MAS and HLH indications. Hemophagocytic Lymphohistiocytosis Competitive Landscape The pipeline of HLH is very scarce, with only three emerging therapies: plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), and tadekinig alfa (r-hIL-18BP) (AB2 Bio). Despite all being in late-stage development, this underscores the scarcity of therapeutic innovation in this space. AB2 Bio's Tadekinig alfa is an innovative recombinant human IL-18 binding protein (IL-18BP) that neutralizes IL-18, a key pro-inflammatory cytokine. Under normal physiological conditions, endogenous IL-18BP is present in excess, maintaining circulating free IL-18 at undetectable levels. In certain autoinflammatory disorders, this regulatory balance is disrupted, leading to elevated systemic free IL-18 and severe pathological hyperinflammation. Tadekinig alfa re-establishes this balance by sequestering excess free IL-18, thereby attenuating inflammatory responses. This mechanism represents a novel and promising therapeutic strategy for autoimmune and inflammatory diseases characterized by elevated systemic IL-18 levels. The therapy is currently being evaluated in a Phase III clinical trial. Electra Therapeutics' ELA026 is a monoclonal antibody developed to treat severe inflammatory diseases driven by dysregulated myeloid cells and T-lymphocytes. It selectively and rapidly depletes the circulating inflammatory myeloid cells and T cells responsible for disease pathology, while preserving the CD47/SIRPα immune checkpoint pathway. ELA026 is presently in Phase II/III clinical development. The anticipated launch of these emerging therapies are poised to transform the hemophagocytic lymphohistiocytosis market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hemophagocytic lymphohistiocytosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about the hemophagocytic lymphohistiocytosis drugs @ Hemophagocytic Lymphohistiocytosis Drugs Market Recent Developments in the Hemophagocytic Lymphohistiocytosis Market In October 2025, Electra Therapeutics announced that the first patients had been dosed in the SURPASS study, a pivotal Phase II/III clinical trial evaluating ELA026, the first investigational therapy designed to broadly treat secondary HLH. In June 2025, Sobi announced that the US FDA approved emapalumab for the treatment of adult and pediatric (newborn and older) patients with HLH/MAS in known or suspected Still's disease, including sJIA, with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. What is Hemophagocytic Lymphohistiocytosis? Hemophagocytic lymphohistiocytosis is a rare, life-threatening immune system disorder characterized by extreme and uncontrolled immune activation. In HLH, immune cells, particularly macrophages and lymphocytes, become overactive and release excessive amounts of inflammatory cytokines, leading to a "cytokine storm" that causes widespread tissue damage and organ failure. The condition can be inherited (primary HLH) due to genetic defects affecting immune regulation, or acquired (secondary HLH) and triggered by infections, malignancies, autoimmune diseases, or other immune challenges. Clinically, HLH often presents with persistent high fever, enlarged liver and spleen, cytopenias, liver dysfunction, and markedly elevated inflammatory markers. Without prompt diagnosis and treatment, HLH can rapidly progress and is frequently fatal. Hemophagocytic Lymphohistiocytosis Epidemiology Segmentation The hemophagocytic lymphohistiocytosis epidemiology section provides insights into the historical and current hemophagocytic lymphohistiocytosis patient pool and forecasted trends for the leading markets. The commonest single disease entities were lymphoma, SLE, and sJIA. There has been evidence of clinically diagnosed HIV, EBV, or CMV infection either in the health record or on the death certificate in 2.3%, 8.9%, and 3.3%, respectively. The hemophagocytic lymphohistiocytosis treatment market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into: Total Incident Cases of HLH Total Incident Cases of HLH by Type Incident Cases of HLH by Gender Incident Cases of Familial HLH by Mutation Incident Cases of Acquired HLH by Etiology Total Treated Cases of HLH Scope of the Hemophagocytic Lymphohistiocytosis Market Report Therapeutic Assessment: Hemophagocytic Lymphohistiocytosis current marketed and emerging therapies Hemophagocytic Lymphohistiocytosis Market Dynamics: Key Market Forecast Assumptions of Emerging Hemophagocytic Lymphohistiocytosis Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Hemophagocytic Lymphohistiocytosis Market Access and Reimbursement Download the report to understand what is the latest research on hemophagocytic lymphohistiocytosis @ New Hemophagocytic Lymphohistiocytosis Medication Table of Contents Related Reports Hemophagocytic Lymphohistiocytosis Clinical Trial Analysis Hemophagocytic Lymphohistiocytosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key HLH companies, including Chia Tai Tianqing Pharmaceutical Group, Bellicum Pharmaceuticals, Atara Biotherapeutics, Incyte Corporation, Alpine Immune Sciences, Expression Therapeutics, among others. Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus 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 SLE companies, including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Clinical Trial Analysis Systemic Lupus Erythematosus Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key SLE companies, including Roche, Biogen, Viatris Inc., Novartis, Qilu Pharmaceutical, ImmuPharma, Beijing Mabworks Biotech Co., Ltd., Zenas Biopharma, AbbVie, Beijing InnoCare Pharma Tech Co., Ltd., Alumis Inc., Galapagos NV, Palleon Pharmaceuticals, Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Johnson & Johnson, Merck KGaA, Resolve Therapeutics, ILTOO Pharma, Argenx, Cartesian Therapeutics, Keymed Biosciences Co.Ltd, BioSenic, Eisai, Vertex Pharmaceuticals, Kyverna Therapeutics, Miltenyi Biomedicine, Astrazeneca, Otsuka Pharmaceutical, Fresh Tracks Therapeutics, among others. Macrophage Activation Syndrome Market Macrophage Activation Syndrome 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 MAS companies, including Electra Therapeutics, TJ Biopharma, AB2 Bio, Novartis, Deepcure, Sobi, 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 21% more press release views with Request a Demo

Drug ApprovalImmunotherapyPhase 3

15 Jan 2026

·www.prnewswire.com

Sobi's 2025 annual revenue and adjusted EBITA margin exceed previous outlook

STOCKHOLM, Jan. 15, 2026 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO: SOBI) announces today that revenue and adjusted EBITA margin for the full year 2025 were higher than previous outlook. Full-year revenue was approximately SEK 28.2 B, representing approximately 15 per cent growth at constant exchange rate (CER)[1]. Adjusted EBITA margin1,[2] was approximately 40 per cent of revenues. The main reason for the increased revenue was higher than expected Q4 sales for Doptelet, Gamifant and the Haemophilia portfolio. The adjusted EBITA margin¹,² was increased due to the higher than expected revenue. At the publication of the Q3 2025 report on 20 October 2025 Sobi stated the outlook for the full year 2025 to be: Revenue was anticipated to grow by a low double-digit percentage at CER¹ and adjusted EBITA margin¹,² was anticipated to be in the mid to high-30s per cent of revenue. Sobi will announce its fourth quarter and full year 2025 report on Thursday 5 February 2026 at 8:00 am CET. Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO: SOBI) is listed on NASDAQ Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 12:30 CET on 15 January 2026. Gerard Tobin Head of Investor Relations [1] Alternative Performance Measures (APMs). [2] Excluding items affecting comparability (IAC). This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB 21% more press release views with Request a Demo

Financial Statement

100 Deals associated with Emapalumab-LZSG

External Link

KEGG	Wiki	ATC	Drug Bank
-	Emapalumab-LZSG	L04AA39	DB14724

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Macrophage Activation Syndrome	United States	NovImmune SA	27 Jun 2025
Systemic onset juvenile chronic arthritis	United States	NovImmune SA	27 Jun 2025
Lymphohistiocytosis, Hemophagocytic	United States	NovImmune SA	20 Nov 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	United States	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Japan	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Belgium	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Canada	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Czechia	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	France	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Germany	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Italy	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Netherlands	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Poland	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05744063 (CTgov)	Phase 4	Lymphohistiocytosis, Hemophagocytic	13	Emapalumab	yeaxcdqdwr = kfhbjxrtvi rnqunmsfcc (egdqbcpbpf, uypxlrnnqn - cliwdtuxoe) View more	-	13 Mar 2026
NCT05001737 (EMERALD \| NI-0501-14, CTgov)	Phase 3	Systemic onset juvenile chronic arthritis \| Fibrous Dysplasia, Polyostotic \| Lymphohistiocytosis, Hemophagocytic ... View more	33	Emapalumab (Cohort 1 (sJIA and AOSD))	nlqbvufvuc = wuqbtpswnp mixlxngwsk (xvwqhsiuwo, liltcuueoa - esshgduatd) View more	-	19 Feb 2026
NCT05001737 (EMERALD \| NI-0501-14, CTgov)	Phase 3		33	Emapalumab (Cohort 2 (SLE))	nlqbvufvuc = bqxmravqwe mixlxngwsk (xvwqhsiuwo, lodepulbxu - wblxkdasnl) View more	-	19 Feb 2026
NCT03311854 (NI-0501-06, ACR2025)	Phase 2	Macrophage Activation Syndrome	39	Emapalumab	vkucqwnvrl(umdzimpggc) = luguhorgrc vauxoutfnk (asdkvxubpz, 37.2 - 69.9) View more	Positive	24 Oct 2025
NCT03311854 (NI-0501-06, ACR2025)	Phase 2	Macrophage Activation Syndrome	39	Emapalumab (EMERALD (NCT05001737))	eqpazsqipn(sozrbbqmdv) = khboinstfs wvaqlzfapa (hffgufqrud ) View more	Positive	24 Oct 2025
NCT03311854 (NI-0501-06, ACR2025)	Phase 2	Macrophage Activation Syndrome	39	Emapalumab (NI-0501-06 (NCT03311854))	vghusurvuj(qcvbxdiccc) = rclopccljr swayusazen (olugnaqnra )	Positive	24 Oct 2025
NCT03311854 \| NCT05001737 (NI-0501-14, FDA_CDER) Manual	Not Applicable	Systemic onset juvenile chronic arthritis \| Lymphohistiocytosis, Hemophagocytic \| Macrophage Activation Syndrome	39	GAMIFANT	fdfrlbzjcg(sgewyvxmsj) = eldzrglcwg qaerroouxt (jjsvcsfzny, 37.2 - 69.9) View more	Positive	27 Jun 2025
NCT03311854 (NI-0501-06, EULAR2025)	Phase 2	Macrophage Activation Syndrome	39	Emapalumab (MAS in Still's disease + Inadequate response to high-dose glucocorticoids)	jfclbmwbzk(tkjaddwhin) = wjpqaguhvj hiqmctuquq (wbqsepzzwy )	Positive	11 Jun 2025
PS2153 (EHA2025)	Not Applicable	Cytokine Release Syndrome IFN-γ \| TNF-α \| IL-2 ... View more	38	Emapalumab	awussgamvp(nuieglqpkc) = 8 patients died (2 from disease relapse/progression, 4 from severe infections, and 2 from septic shock associated with CRS) paplmyjgbz (npohoupeom ) View more	Positive	14 May 2025
NCT03311854 (NI-0501-06, ACR2024)	Phase 3	Cardiovascular Diseases	39	Emapalumab	ocadgciwhe(avylnduydo) = xczsvmbudy rworprqaqm (saenqotbfc, 37.2 - 69.9) View more	Positive	10 Nov 2024
NCT04731298 (P449, CTgov)	Phase 2	Graft dysfunction	2	Emapalumab (Emapalumab Treated)	rkzdltyxdx = jxhdbsrero pfaztroebl (zebfnuwbqn, ugnuijoyhh - zquyoookep) View more	-	28 Dec 2023
NCT04731298 (P449, CTgov)	Phase 2	Graft dysfunction	2	emapalumab (Non-emapalumab Treated)	rkzdltyxdx = qsantxtdbm pfaztroebl (zebfnuwbqn, ijmxiqwynm - iaenfvusul) View more	-	28 Dec 2023
NCT04765553 (CTgov)	Phase 1	Rare Diseases	8	NI-0501 (Emapalumab)	bmuctuvjfk(aqwadjggwh) = zxvbwzhwgd jwmmtiwfae (kbxvaytcaa, ibqxxizzkt - vibtbywubc) View more	-	24 Nov 2023
NCT04765553 (CTgov)	Phase 1	Rare Diseases	8	Saline (Placebo)	fdxqprgsph = tocawdvsow iqpgpmtvhc (ooxwpghcjd, fejrrboyyr - gmyzjnhybl) View more	-	24 Nov 2023

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