RegulationBreakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Priority Review (Australia)

Structure/Sequence

Molecular FormulaC29H36N6O6

InChIKeyPLILLUUXAVKBPY-SBIAVEDLSA-N

CAS Registry496775-62-3

249

Clinical Trials associated with Eltrombopag Diolamine

TCTR20250910007

/ Not yet recruitingPhase 1

A Single Dose, Randomized, Open-label, Two-way Crossover Bioequivalence Study of Generic Eltrombopag Olamine 25 mg Film-coated Tablets and Reference Product (Revolade) in Healthy Thai Volunteers under Fasting Conditions

Start Date29 Apr 2026

Sponsor / Collaborator-

TCTR20240925001

/ Not yet recruitingPhase 1IIT

A Bioequivalence study of a randomized, open-label, single dose, two-way crossover design with two-period, two-treatment, and two-sequence of Eltrombopag 25 mg tablets relative to Revolade 25 mg tablets in healthy Thai volunteers under fasting condition.

Start Date29 Jan 2026

Sponsor / Collaborator

Bio-innova Co., Ltd

NCT07189910

/ Not yet recruitingPhase 4IIT

Efficacy and Safety of Glucocorticoids Combined With Recombinant Human Thrombopoietin (rhTPO) Sequential Eltrombopag Olamine for Severe Primary Immune Thrombocytopenia (ITP) in Adults

To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. In this study, hospitalized participants will receive a 14-day regimen of high-dose dexamethasone (HD-DXM) plus recombinant human thrombopoietin (rhTPO), followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP patients.

Start Date01 Dec 2025

Sponsor / Collaborator

Wuhan Xiehe Hospital Tower

100 Clinical Results associated with Eltrombopag Diolamine

100 Translational Medicine associated with Eltrombopag Diolamine

100 Patents (Medical) associated with Eltrombopag Diolamine

1,530

Literatures (Medical) associated with Eltrombopag Diolamine

31 Dec 2025·Hematology

Sustained response off treatment after fostamatinib in refractory immune thrombocytopenia: A series of four case reports

Article

Author: Ghanima, Waleed ; Rackwitz, Stefan ; Lucas Boronat, Francisco Javier ; Carrai, Valentina

INTRODUCTION:

A goal of most primary immune thrombocytopenia (ITP) treatments is reducing or discontinuing treatment while maintaining a response including an absence of bleeding events. We present four cases describing treatment with the spleen tyrosine kinase (SYK) inhibitor, fostamatinib, that showed sustained response off treatment (SROT).

CASE PRESENTATIONS:

Case 1 was a 66-year-old male with chronic ITP. He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo). He received fostamatinib in the FIT-3 open-label extension for seven weeks and maintained SROT for 2.5 years. Case 2 was a 54-year-old female patient with chronic, highly refractory ITP. SROT was achieved after 6 months of fostamatinib and was maintained for more than 16 months (in remission to date). Case 3 was a 60-year-old male with chronic ITP. He was successfully treated with cycles of corticosteroids for six years prior to fostamatinib. He was treated with fostamatinib plus prednisone for approximately two months. SROT was observed in this patient for one year. Case 4 was a 67-year-old male with persistent ITP. Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim. After 11 months of fostamatinib, his dose was tapered for three months and ultimately discontinued. SROT was observed for more than ten months (in remission to date).

DISCUSSION:

These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT.

31 Dec 2025·PLATELETS

A case with a novel GATA1 variant mimicking immune thrombocytopenia attacks

Article

Author: Aktekin, Elif Habibe ; Ceylaner, Serdar ; Erbay, Ayşe ; Beşen, Şeyda ; Yazıcı, Nalan

One of the leading thrombocytopenia occasions in childhood is immune thrombocytopenia (ITP). Small number of these patients do not respond to treatment methods known to be effective for ITP such as corticosteroids, intravenous immunoglobulin and thrombopoietin receptor agonists (eltrombopag, romiplostim, etc.) and have serious bleeding symptoms. We present a case in which a molecular genetic analysis was performed due to challenging responses to medical treatment for severe systemic bleeding symptoms which were similar to ITP. A hemizygous variant was detected in the GATA1 gene and a homozygous variant in the ATP2A1 gene for Brody myopathy. Eltrombopag, romiplostim, IVIG and corticosteroid were applied to resistant thrombocytopenia of the patient without knowing the cause. Patient had response in platelet counts in some occasions. This situation attracted our attention after the detection of this GATA variant in the etiopathogenesis of thrombocytopenia. Further investigations should be performed for much more rare causes of thrombocytopenia in patients with refractory thrombocytopenia attacks.

31 Dec 2025·Virulence

Effectiveness of antiviral, immunomodulatory and platelet-enhancing agents for treatment of dengue infection: A systematic review

Review

Author: Aynekulu Mersha, D.G. ; Goeijenbier, M. ; Duijff, F. ; Martina, B. ; Dalm, V.A.S.H. ; van Gorp, E.C.M. ; Langerak, T. ; Hakim, M.S. ; van Lelyveld, S.F.L.

Dengue is a vector-borne infectious disease, caused by dengue virus (DENV), with a rapidly increasing incidence worldwide. With no feasible, widely applicable prevention method available in the near term, the need for an effective treatment is of great importance. This systematic review aims to provide a comprehensive overview of potential antiviral, immunomodulatory, and platelet-enhancing therapies for the treatment of DENV. This systematic review was conducted according to the PRISMA guidelines. Clinical trials that investigate treatment options for DENV in the general population were included. Twenty-six studies were included, investigating length of hospital stay (n = 10), platelet count (n = 16), interleukin (IL)-6 levels (n = 4), virological log reduction (VLR) (n = 2), and non-structural (NS)-1 clearance time (n = 4). Focusing on potential antiviral agents, four studies showed a significant reduction regarding length of hospital stay, of which two used doxycycline. The most profound reduction of hospital stay was observed when doxycycline was combined with Carica papaya herbal extract (7.3 days vs 9.1 days). This combination was also able to achieve a significant rise in platelet count (+154.1 × 10⁹/L vs + 66.0 × 10⁹/L in 7 days). Immunomodulatory therapies did not demonstrate efficacy against DENV, although some evidence suggests that rupatadine may increase platelet count. The platelet-enhancing agents recombinant human IL-11, anti-rhD immunoglobulin (anti-D), and eltrombopag all showed a significant rise in platelet count. Small sample sizes make it challenging to draw definitive conclusions out of the included studies. Larger clinical trials are needed to evaluate treatments for DENV, with particular focus on doxycycline, Carica papaya, rupatadine, and platelet-enhancing agents.

News (Medical) associated with Eltrombopag Diolamine

17 Dec 2025

·www.drugs.com

American Society of Hematology, Dec. 6 to 9

The annual meeting of the American Society of Hematology was held from Dec. 6 to 9 in Orlando, Florida, and attracted participants from around the world, including hematology specialists as well as clinical practitioners and other health care professionals. The conference featured presentations focusing on the diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems. In the phase 3 GIMEMA ALL2820 trial, Sabina Chiaretti, M.D., Ph.D., of Sapienza University in Rome, and colleagues found a chemo-free targeted/immunotherapy-based approach to be significantly beneficial for patients with newly diagnosed Ph+ acute lymphoblastic leukemia (ALL) and found that it outperformed a tyrosine kinase inhibitor/chemotherapy-based backbone. The authors conducted a randomized trial (2:1 ratio in favor of the chemo-free arm) designed for newly diagnosed Ph+ ALL patients (older than 18 years; no upper age limit), in which the chemo-free arm was based on induction with ponatinib (45 or 30 mg daily, depending on age) followed by at least two cycles of intravenous blinatumomab (maximum, five cycles). The control arm was based on the administration of imatinib and chemotherapy (again, dose adjusted according to age). A crossover was allowed from the control to the experimental arm in the case of molecular residual disease persistence or development of ABL1 mutations. The researchers observed a significant advantage of the experimental arm over the control arm, with hematologic responses at the end of induction being 94.3 and 79.4 percent, respectively, while death was 2.5 and 10.2 percent, respectively, and off-treatment was 2.8 and 8.9 percent, respectively. In line with the findings, molecular responses after consolidation (two cycles of blinatumomab/four to six cycles of chemotherapy) were significantly better in the experimental arm (70.9 versus 48.7 percent); relapses occurred slightly less frequently in the experimental arm (6 versus 8 percent). Treatment was well tolerated overall. Event-free survival was 90 and 74 percent and overall survival was 94 and 77 percent in the experimental and control arms, respectively. Notably, patients undergoing crossover had an overall survival of 97 percent. "These findings should be regarded as clinical practice-changing. Nonetheless, most countries do not have access to blinatumomab in the first-line setting," Chiaretti said. "Thus, there is a strong wish that these results will lead to blinatumomab approval, not only for patients enrolled in the clinical trials, but for all patients." Several authors disclosed ties to pharmaceutical and biotechnology companies, including Amgen (blinatumomab) and Takeda (ponatinib). Abstract No. 439 In the phase 3 VAYHIT2 study, Hanny Al-Samkari, M.D., of Massachusetts General Hospital in Boston, and colleagues found that a monoclonal antibody that inhibits the B-cell activating factor receptor ianalumab improves outcomes among patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids. The investigators randomly assigned (1:1:1) 152 patients with primary ITP to receive eltrombopag plus either ianalumab 9 mg/kg or 3 mg/kg or placebo. The primary end point was time to treatment failure (TTF), and the key secondary end point was stable response at six months (SR6). The researchers found that TTF was significantly longer with ianalumab 9 mg/kg and ianalumab 3 mg/kg when compared with placebo. Significantly more patients achieved SR6 with ianalumab 9 mg/kg compared with placebo. Furthermore, response and complete response rates were significantly higher with ianalumab 9 mg/kg. "Ianalumab in combination with eltrombopag prolonged TTF, improved SR6, reduced fatigue, facilitated tapering off eltrombopag, and delayed need for subsequent therapy in patients with primary ITP previously treated with corticosteroids," the authors write. "Ianalumab was well tolerated, with no observed increase in infection risk relative to placebo. Ianalumab may be disease-modifying when used early in the course of ITP." Several authors disclosed ties to pharmaceutical and biotechnology companies, including Novartis, the manufacturer of ianalumab. Abstract No. LBA-2 In a phase 3 study, Wojciech Jurczak, M.D., Ph.D., of the National Research Institute of Oncology in Krakow, Poland, and colleagues found that the noncovalent Bruton tyrosine kinase inhibitor pirtobrutinib improves outcomes among patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The authors assessed the efficacy and safety of pirtobrutinib compared with bendamustine plus rituximab (BR) in patients with CLL/SLL who had not undergone treatment. For the study, 282 patients were randomly assigned (1:1) to receive either pirtobrutinib monotherapy or six cycles of BR. The researchers found that independent review committee progression-free survival (the primary end point) was significantly improved with pirtobrutinib compared with BR. Pirtobrutinib was found to be well tolerated. Specifically, the investigators identified only low discontinuation rates and the presence of atrial fibrillation/flutter. "While overall survival data remained immature, a notable trend favoring pirtobrutinib was observed, despite 52.9 percent of BR patients crossing over to receive pirtobrutinib after disease progression," the authors write. "Taken together, these data suggest that pirtobrutinib may be considered a potential new standard-of-care treatment for patients with untreated CLL/SLL, including older patients who may receive only one line of therapy." Several authors disclosed ties to pharmaceutical and biotechnology companies, including Eli Lilly, the manufacturer of pirtobrutinib. Abstract No. LBA-3 ASH: Financial Difficulties Common During Pediatric Leukemia Treatment MONDAY, Dec. 15, 2025 -- Nearly one-third of families of children receiving chemotherapy for acute lymphoblastic leukemia develop serious financial difficulties during their child's treatment, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Iron Overload Prevalent in Sickle Cell Disease WEDNESDAY, Dec. 10, 2025 -- Iron overload is prevalent among adults with sickle cell disease, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Late Effects Uncommon for Patients With Sickle Cell Disease After Allogeneic HCT WEDNESDAY, Dec. 10, 2025 -- Late effects are rare for patients with sickle cell disease undergoing allogeneic hematopoietic cell transplantation, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Sustained Benefit Seen for Etranacogene Dezaparvovec in Hemophilia B WEDNESDAY, Dec. 10, 2025 -- For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five years, according to a study published online Dec. 7 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Free Full Text ASH: Agent Orange Exposure Linked to Increased Risk for Myelodysplastic Syndrome TUESDAY, Dec. 9, 2025 -- Agent orange exposure is associated with an increased risk for myelodysplastic syndrome, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Lifestyle Intervention Feasible During Chemo for Lymphoma TUESDAY, Dec. 9, 2025 -- A lifestyle intervention is feasible for patients with lymphoma undergoing chemotherapy, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Few Sickle Cell ED Visits Adhere to Guidelines for Opioid Pain Meds TUESDAY, Dec. 9, 2025 -- For patients with acute sickle cell disease pain presenting to the emergency department, guideline adherence for delivery of opioid pain medications is inadequate, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: No Specific Harms Found for Hydroxyurea Use During Pregnancy TUESDAY, Dec. 9, 2025 -- There is no clear evidence of harm for women receiving hydroxyurea during pregnancy for sickle cell disease, according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text ASH: Guidelines Updated for Management of Acute Myeloid Leukemia in Older Adults MONDAY, Dec. 8, 2025 -- In updated 2025 American Society of Hematology guidelines, recommendations are presented for the management of newly diagnosed acute myeloid leukemia in older adults; the guidelines were published online Nov. 25 in Blood Advances to coincide with the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida. Read Full Text

Clinical ResultPhase 3ASH

12 Dec 2025

·www.drugs.com

Experimental Drug, Ianalumab, Provides Lasting Relief For Bleeding Condition

FRIDAY, Dec. 12, 2025 — More than half of people with a rare life-threatening bleeding condition received lasting relief from an experimental antibody treatment, clinical trial results show. Ianalumab, a monoclonal antibody, helped patients with immune thrombocytopenia (ITP) maintain safe platelet counts without serious bleeding episodes for at least a year, according to results published Dec. 9 in The New England Journal of Medicine . The drug achieved that after four monthly doses, researchers reported. “This study shows that prolonged, durable responses to ITP treatment, without the need for ongoing therapy, are possible — and that’s a huge advantage for patients,” lead researcher Dr. Adam Cuker , clinical director of the University of Pennsylvania Blood Disorders Center, said in a news release. ITP is an autoimmune disease that causes the body’s immune cells to attack platelets, the blood cells responsible for clotting, researchers said in background notes. It affects about 50,000 people in the U.S. The disease causes abnormal bleeding from the skin and mucous membranes, including nosebleeds, gum bleeding and heavy menstruation. Patients might also suffer from easy bruising and fatigue. ITP patients who develop low platelet counts or suffer severe bleeding initially are treated with steroids, researchers said. After that, the U.S. Food and Drug Administration (FDA) has approved three drugs for ITP, but all require that people take daily pills or weekly injections for the rest of their lives. “As a hematologist, I’m glad that we have effective therapies for ITP, but they’re not necessarily ideal for chronic disease management or long-term quality of life,” Cuker said. Ianalumab works by targeting the immune cells that generate platelet-attacking antibodies, researchers said. For the new trial, researchers recruited 152 people with ITP and randomly assigned them to one of three groups. The first two groups got either a high or low dose of ianalumab, while the third group received a placebo. The patients received their doses once a month for four months. Everyone also received eltrombopag , a drug that spurs platelet production, for up to four months. Results showed that 54% of people who got a higher dose of ianalumab and nearly 51% of those who got the lower dose were likely to maintain normal platelet counts for a year, compared to only 30% of those receiving a placebo. When platelet counts were measured at six months — two months after the last dose of ianalumab — 62% of the high-dose patients had stable platelet counts, versus 39% in the placebo arm. Additional clinical trials for ianalumab are ongoing, including studies of other autoimmune disorders, researchers said. Researchers will continue to follow the patients from this trial to track their long-term response. "We’re looking forward to seeing if the treatment-free responses in this study extend out even further,” Cuker said. “Improving the long-term reality of living with ITP is not something we’ve been able to think about before. The goal has always been to improve platelet counts or reduce the risk of bleeding, but this research is ushering in a new era of hope for patients with ITP.” Ianalumab is not yet FDA-approved for patients, researchers noted. Novartis , the developer of ianalumab, funded the clinical trial. Sources University of Pennsylvania, news release, Dec. 9, 2025

Clinical ResultClinical Study

09 Dec 2025

·www.globenewswire.com

Novartis ianalumab significantly extends disease control in patients with immune thrombocytopenia with only four once-monthly doses

Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45% with patients maintaining disease control 2.8 times longer than with placebo plus eltrombopag1,2 62% of patients treated with ianalumab plus eltrombopag achieved sustained platelet response at six months compared to 39% with placebo plus eltrombopag1,2Ianalumab, administered as four once-monthly intravenous doses in the ITP setting, has the potential to reduce the need for chronic treatment and deliver durable disease control in ITPNovartis plans to submit VAYHIT2 data from second-line ITP with results from ongoing first-line ITP trial, VAYHIT1, to health authorities in 2027 Basel, December 9, 2025 – Novartis today announced positive results from VAYHIT2, a Phase III trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1-3. Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45%, based on the primary endpoint of time to treatment failure (TTF), which assesses how long patients maintain safe platelet levels during and after the treatment period1,2. The median time to treatment failure for patients receiving ianalumab plus eltrombopag was 2.8 times longer than those on placebo plus eltrombopag (13.0 months vs. 4.7 months)1,2.Detailed data will be presented during the Late-Breaking Abstract Session at the 67th American Society of Hematology Annual Meeting & Exposition (ASH) and simultaneously published in The New England Journal of Medicine1,2.“Treatments for ITP have historically focused on raising platelet counts, often requiring chronic therapy to control ITP. This means many patients remain on treatment long-term, facing persistent disease burden and symptoms like fatigue,” said Hanny Al-Samkari, M.D., Peggy S. Blitz Endowed Chair in Hematology/Oncology, Mass General Brigham, and Associate Professor of Medicine, Harvard Medical School. “The VAYHIT2 trial results are encouraging, as they demonstrated improved disease control even while patients spend time off treatment, pointing toward possible progress for people living with ITP.”Patients receiving ianalumab (9 mg/kg) plus eltrombopag also achieved a significantly higher rate of sustained platelet count improvement at six months versus placebo plus eltrombopag (62% vs. 39%), meeting the key secondary endpoint1,2. Fatigue improvement, as measured by PROMIS Fatigue, showed a mean reduction of 7.7 points with ianalumab plus eltrombopag versus 3.6 points with placebo plus eltrombopag1,2.“B cells drive the autoimmune response that leads to platelet destruction and increased bleeding risk in ITP. The novel dual mechanism of action of ianalumab aims to deplete B cells while blocking their survival signals,” said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. “Guided by our decades-long experience advancing ITP care, the VAYHIT2 findings underscore the potential of ianalumab to deliver durable control with a short course of four once-monthly doses, offering patients the possibility of achieving disease stability without ongoing treatment.”Two doses of ianalumab were assessed in VAYHIT2 with ianalumab 9 mg/kg demonstrating statistically significant improvements across both the primary and key secondary endpoints, and ianalumab 3 mg/kg demonstrating statistically significant improvements in the primary endpoint and numerical improvements in the key secondary endpoint1-3. Ianalumab 9 mg/kg + eltrombopag (N=50) Ianalumab 3 mg/kg + eltrombopag (N=51) Placebo + eltrombopag (N=51) Primary endpoint: Time to treatment failure (TTF) 13.0 months (HR 0.55; 95% CI: 0.32, 0.92; p=0.021a) Not estimable (HR 0.58; 95% CI: 0.34, 0.98; p=0.023a) 4.7 months Key secondary endpoint: Stable response at 6 months (SR6) 62.0% (p=0.023a) 56.9% (p=0.035a) 39.2% a. Required p-value for statistical significance is one-sided <0.025Ianalumab was well tolerated with no new safety signals, and the side effect profile was consistent with previous studies1,2. Adverse events were comparable between the ianalumab and placebo arms, with the most common AEs for ianalumab plus eltrombopag being headache (14% with 9 mg/kg, 10% with 3 mg/kg vs. 8% with placebo) and infusion-related reactions (14% with 9 mg/kg, 8% with 3 mg/kg vs. 8% with placebo)1,2. Neutropenia* occurred more frequently in the ianalumab groups (16% with 9 mg/kg, 12% with 3 mg/kg) compared to placebo (2%) with most cases resolving without requiring treatment or dose interruption1,2. No on-treatment adverse event led to permanent discontinuation of therapy1,2. VAYHIT2 marks the third positive Phase III trial with ianalumab, following two positive trials in adults with active Sjögren's disease1,4. Novartis plans to submit the data from VAYHIT2 along with results from the ongoing first-line ITP trial, VAYHIT1, in 2027. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency5,6. *An adverse event of special interest encompassing several terms related to low levels of neutrophils, neutrophil precursors and leukocytesAbout ianalumab Ianalumab (VAY736) is a novel fully human monoclonal antibody being investigated for its potential to treat various B cell-driven autoimmune diseases, including Sjögren’s disease, immune thrombocytopenia (ITP), systemic lupus erythematosus (SLE), lupus nephritis (LN), warm autoimmune hemolytic anemia (wAIHA) and diffuse cutaneous systemic sclerosis (dcSSc)3,7-13. Its mechanism of action targets B cells in two ways, namely combining B cell depletion via antibody-dependent cellular toxicity (ADCC) and interruption of BAFF-R mediated signals of B cell function and survival8. In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren’s disease, systemic lupus erythematosus, and immune thrombocytopenia4,14-16. Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 202417. About primary immune thrombocytopenia Primary immune thrombocytopenia (ITP) is a rare, autoimmune disorder in which the immune system mistakenly targets and destroys platelets, the cells essential for blood clotting18. This can lead to symptoms such as prolonged bleeding, easy bruising and chronic fatigue, which can significantly impact daily life18,19. Despite available treatments, many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control20. Current options often focus on maintaining safe platelet levels and preventing bleeding complications and may require ongoing use20,21. The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life19,22. There is a need for therapies that offer durable response while reducing the burden of long-term treatment23. About VAYHIT2 VAYHIT2 (NCT05653219) is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of two different doses of ianalumab versus placebo, in addition to eltrombopag, in adults with primary immune thrombocytopenia (ITP) (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids3. Alongside eltrombopag, patients were randomized 1:1:1 to receive four once-monthly intravenous infusions of ianalumab at 3 mg/kg, ianalumab at 9 mg/kg or placebo3. The primary endpoint was time to treatment failure, which is defined as the time from randomization until either: a platelet count of less than 30 G/L later than 8 weeks from randomization; the need for rescue therapy later than 8 weeks from randomization; initiation of a new ITP treatment at any time; ineligibility or inability to taper/discontinue eltrombopag; or death3. The key secondary endpoint is the percentage of patients with a stable platelet count response at Month 63. Other secondary endpoints include measures of depth and duration of platelet response as well as patient-reported outcomes that measure quality of life and fatigue, among other endpoints3. DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.References Primary results from VAYHIT2, a randomized, double-blind, phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatment. Oral presentation at ASH Annual Meeting & Exposition: December 6-9, 2025.Cuker A, Stauch T,Cooper N et al. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. DOI: 10.1056/NEJMoa2515168Clinicaltrials.gov. NCT05653219. A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids (VAYHIT2). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05653219Thomas GB, Xavier M, Stephanie F et al. Ianalumab demonstrates significant reduction in disease activity in patients with Sjögren’s Disease: Efficacy and safety results from two global Phase 3, randomized, placebo-controlled double-blind studies (NEPTUNUS-1 and NEPTUNUS-2). Presented at the American College of Rheumatology (ACR) Congress; October 24-29, 2025; Chicago, Illinois.US Food and Drug Administration. Orphan drug designation: ianalumab—treatment of primary immune thrombocytopenia. Published February 13, 2025. Accessed November 14, 2025. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=1018924European Commission. Community register of orphan medicinal products: ianalumab. Updated October 14, 2025. Accessed November 14, 2025. https://ec.europa.eu/health/documents/community-register/html/o3036.htmClinicaltrials.gov. NCT05648968. A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia (VAYHIA) Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05648968Dörner T, Bowman SJ, Fox R, et al. Safety and Efficacy of Ianalumab in Patients With Sjögren's Disease: 52-Week Results From a Randomized, Placebo-Controlled, Phase 2b Dose-Ranging Study. Arthritis Rheumatol. 2025;77(5):560-570. doi:10.1002/art.43059Clinicaltrials.gov. NCT05350072. Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-1). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05350072Clinicaltrials.gov. NCT05349214. Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-2). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05349214Clinicaltrials.gov. NCT05639114. Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1) (SIRIUS-SLE 1). Accessed November 14, 2025. https://www.clinicaltrials.gov/study/NCT05639114Clinicaltrials.gov. NCT05126277. Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis (SIRIUS-LN). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05126277Clinicaltrials.gov. NCT06470048. A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis. Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT06470048Bowman SJ, Fox R, Dörner T, et al. Safety and efficacy of subcutaneous ianalumab (VAY736) in patients with primary Sjögren's syndrome: a randomised, double-blind, placebo-controlled, phase 2b dose-finding trial. Lancet. 2022;399(10320):161-171. doi:10.1016/S0140-6736(21)02251-0Shen N, Ignatenko S, Gordienko A, et al. Phase 2 Safety and Efficacy of Subcutaneous (s.c.) Dose Ianalumab (VAY736; Anti-BAFFR mAb) Administered Monthly over 28 Weeks in Patients with Systemic Lupus Erythematosus (SLE) of Moderate-to-Severe Activity [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 9). Accessed November 14, 2025. https://acrabstracts.org/abstract/phase-2-safety-and-efficacy-of-subcutaneous-s-c-dose-ianalumab-vay736-anti-baffr-mab-administered-monthly-over-28-weeks-in-patients-with-systemic-lupus-erythematosus-sle-of-moderate-to-severe/Bradbury C, Elverdi T, Trautmann K, et al. A phase 2 study of ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapy (VAYHIT3). HemaSphere. 2025;9(Suppl 1):Abstract S238. Presented at European Hematology Association (EHA) Congress, June 12‑15, 2025. Milan, Italy. Accessed November 14, 2025. https://library.ehaweb.org/eha/2025/eha2025-congress/4159389/charlotte.bradbury.a.phase.2.study.of.ianalumab.in.patients.with.primary.htmlNovartis. Press release. Novartis to strengthen oncology pipeline with agreement to acquire MorphoSys AG for EUR 68 per share or an aggregate of EUR 2.7bn in cash. February 5, 2024. Accessed November 14, 2025. https://www.novartis.com/news/media-releases/novartis-strengthen-oncology-pipeline-agreement-acquire-morphosys-ag-eur-68-share-or-aggregate-eur-27bn-cashRodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386-2393. doi:10.1182/blood-2008-07-162503Kuter DJ, Mathias SD, Rummel M, et al. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012;87:558-61Kuter DJ. The treatment of immune thrombocytopenia (ITP)—focus on thrombopoietin receptor agonists. Ann Blood. 2021;6:27. doi:10.21037/aob-2021-itp-04Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875-887. doi:10.1007/s12325-015-0240-zCooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021;96(2):199-207. doi:10.1002/ajh.26083Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, et al. Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022;15(7):779. doi:10.3390/ph15070779 # # # Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

Clinical ResultPhase 2Phase 3ASHOrphan Drug

100 Deals associated with Eltrombopag Diolamine

External Link

KEGG	Wiki	ATC	Drug Bank
-	Eltrombopag Diolamine	B02BX05	DB06210

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Heparin-induced thrombocytopenia	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	12 Sep 2024
Anemia, Aplastic	European Union	Novartis Europharm Ltd.	11 Mar 2010
Anemia, Aplastic	Iceland	Novartis Europharm Ltd.	11 Mar 2010
Anemia, Aplastic	Liechtenstein	Novartis Europharm Ltd.	11 Mar 2010
Anemia, Aplastic	Norway	Novartis Europharm Ltd.	11 Mar 2010
Purpura, Thrombocytopenic, Idiopathic	European Union	Novartis Europharm Ltd.	11 Mar 2010
Purpura, Thrombocytopenic, Idiopathic	Iceland	Novartis Europharm Ltd.	11 Mar 2010
Purpura, Thrombocytopenic, Idiopathic	Liechtenstein	Novartis Europharm Ltd.	11 Mar 2010
Purpura, Thrombocytopenic, Idiopathic	Norway	Novartis Europharm Ltd.	11 Mar 2010
Thrombocytopenia	United States	Novartis Pharmaceuticals Corp.	20 Nov 2008

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Solid tumor	Phase 3	Taiwan Province	Novartis AG	01 Mar 2014
Contusions	Phase 3	United States	GSK Plc	01 Mar 2012
Contusions	Phase 3	Argentina	GSK Plc	01 Mar 2012
Contusions	Phase 3	Czechia	GSK Plc	01 Mar 2012
Contusions	Phase 3	Germany	GSK Plc	01 Mar 2012
Contusions	Phase 3	Hong Kong	GSK Plc	01 Mar 2012
Contusions	Phase 3	Israel	GSK Plc	01 Mar 2012
Contusions	Phase 3	Italy	GSK Plc	01 Mar 2012
Contusions	Phase 3	Poland	GSK Plc	01 Mar 2012
Contusions	Phase 3	Russia	GSK Plc	01 Mar 2012

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05653219 (VAYHIT2, N Engl J Med \| Company_Website) Manual	Phase 3	Purpura, Thrombocytopenic, Idiopathic Second line	152	Ianalumab 9 mg + Eltrombopag	yehpcbsuas(xxeyxjlbaq) = wmtsxcwvwo coftfrqjwu (dcwrxjzemd, 39 - 67) View more	Positive	09 Dec 2025
				Ianalumab 3 mg + Eltrombopag	yehpcbsuas(xxeyxjlbaq) = ujblfrjdtx coftfrqjwu (dcwrxjzemd, 36 - 64) View more
NCT05653219 (VAYHIT2, Pubmed \| N Engl J Med)	Phase 3	Purpura, Thrombocytopenic, Idiopathic Second line	152	Ianalumab 9 mg	hincdryves(qyvnxrltbl) = isllehylrm vnbpmapqyu (upoxakixto ) View more	Positive	09 Dec 2025
				Ianalumab 3 mg	sdygbzlcgm(mrzwzvcmmf) = sqsoyztrid oogirknxqs (icmcjuuzed, 36 - 64) View more
NCT03939637 (PINES, ASH2025)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	118	Eltrombopag	crxaztzvyw(vurtxjxfcg) = mhuyfycmdr kcdogxnbla (diyyfawdwg ) View more	Positive	06 Dec 2025
				Standard of Care (SOC)	crxaztzvyw(vurtxjxfcg) = txifecxljr kcdogxnbla (diyyfawdwg ) View more
ASH2025	Not Applicable	Purpura, Thrombocytopenic, Idiopathic First line	103	Dexamethasone and Eltrombopag (DE)	wypenlsnxn(tjnbxorzde) = xpylfjsntl hkzbhpeyjw (hzwttkqxle ) View more	Positive	06 Dec 2025
				Dexamethasone, Eltrombopag and Azathioprine (DEA)	wypenlsnxn(tjnbxorzde) = lirypeokgj hkzbhpeyjw (hzwttkqxle ) View more
NCT04304820 (ASH2025)	Phase 2	Refractory Severe Aplastic Anemia	45	Low dose CSA (2mg/kg daily) + full dose EPAG	gmqfjclncv(fpcjpzshwh) = dapjueoyor vamegufqop (auhnglxqnm ) View more	Positive	06 Dec 2025
				hATG/CSA/EPAG	zzkeggwzlj(ccgpijtnya) = dtlaxcausn gngdmeprrt (ejsxzrwott ) View more
ASH2025	Not Applicable	Anemia, Aplastic	118	Eltrombopag+cyclosporin A	ormiqwjtjg(btezhgeows) = jqgzxvswop ziewpgvppy (ykwxdjbosc ) View more	Positive	06 Dec 2025
				Eltrombopag+cyclosporin A+antithymocyte globulin	ormiqwjtjg(btezhgeows) = mehzdjkiai ziewpgvppy (ykwxdjbosc ) View more
ASH2025	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	3,710	Eltrombopag	oznsoockqt(vykcssytxy): HR = 1.93 (95.0% CI, 1.11 - 3.34)	Positive	06 Dec 2025
				Romiplostim
ASH2025	Not Applicable	Thrombocytopenia	6	Eltrombopag	pohtgzuoim(jkranlzxsa) = No grade ≥3 bleeding events, thrombotic complications, or clonal evolution were observed. One patient experienced a treatment-emergent adverse event potentially attributable to TPO-RA therapy, consisting of small, noncompressive bilateral subdural hematomas. qanvyyhjpo (olyxlzefrx )	Positive	06 Dec 2025
				Romiplostim
ASH2025	Not Applicable	Refractory Severe Aplastic Anemia	1,122	Eltrombopag + standard immunosuppressive therapy	onimdwnwig(dcyxxhupmy) = higher in the IST-alone arm, as surrogated by Hgb levels >10 mg/dL (OR= 1.67, 95% CI 1.281-2.178), ANC >1000/uL (OR= 0.718, 95% CI 0.543-0.949), and platelet count >100,000/uL (OR= 2.383 (95% CI 1.873-3.032) at 12 months. epvwsldhjh (ubrqwiiscx ) View more	Positive	06 Dec 2025
				Standard immunosuppressive therapy
ASH2025	Not Applicable	Thrombocytopenia	3,500	Eltrombopag	vgcwlbwibi(kullagzpuv) = n=124 clyqskdiyz (civqpwowfd ) View more	Positive	06 Dec 2025
				Romiplostim

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