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

234

Clinical Trials associated with Eltrombopag Diolamine

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

NCT07059910

/ Not yet recruitingNot ApplicableIIT

Prevalence of Thyroid Disorders in Patients With Immune Thrombocytopenic Purpura and Its Impact on Treatment

Prevalence of thyroid disorders in patients with immune thrombocytopenic purpura and its impact on treatment

Start Date01 Jul 2025

Sponsor / Collaborator

Assiut University

NCT06630221

/ RecruitingPhase 2IIT

A Phase II Study of Eltrombopag as a Novel Therapeutic Approach for Patients With Low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) With TET2 Mutations

The purpose of this study is to evaluate if a study drug called eltrombopag can improve the blood cell counts in patients with low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) with mutations in TET2 gene, observe changes in the TET2 gene over time, and evaluate the effectiveness of the treatment. TET2 gene is one of the most frequently mutated genes (altered parts of the DNA) in MDS and CMML.
Eltrombopag is a Food and Drug Administration (FDA) approved drug for the treatment of severe aplastic anemia and low levels of platelets in patients with persistent or chronic immune thrombocytopenia (ITP) and chronic hepatitis C. Eltrombopag is considered investigational (experimental) in this study because the FDA has not approved its use in the treatment of low-risk MDS or CMML. Eltrombopag is a drug that helps stimulate the body's process of making more platelets (small components of blood that help with clotting) by interacting with specific parts of cells. This interaction starts a series of signals that encourage the growth and development of the cells that produce platelets. It was found that this drug could stop the growth of TET2 mutated cells.

Start Date17 Jun 2025

Sponsor / Collaborator

The Case Comprehensive Cancer Center

100 Clinical Results associated with Eltrombopag Diolamine

100 Translational Medicine associated with Eltrombopag Diolamine

100 Patents (Medical) associated with Eltrombopag Diolamine

1,682

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.

01 Nov 2025·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Effect of eltrombopag on biochemical assay parameters

Article

Author: Yamashita, Naoki ; Goto, Hideki ; Kojima, Kazushige ; Teshima, Takanori ; Seimiya, Masanori ; Nakano, Keiichi

Eltrombopag, which is used to treat thrombocytopenia, causes bilirubin-like yellowish-brown serum discoloration and interferes with biochemical parameter measurements. However, the extent of these interferences and the methods and reagents involved remain unclear. Although previous studies have examined the effects of eltrombopag on bilirubin measurements, its effect on enzymatic measuring of bilirubin levels, which is widely used in Japan, has been limited. Serum samples were collected from 17 eltrombopag-receiving patients. Eltrombopag was added to drug-free serum samples at concentrations ranging from 2.5 to 40.0 µg/mL. The light absorption spectra of the samples were recorded. The lipemia index and 21 biochemical parameters, including total bilirubin (TB) levels, measured by enzymatic, vanadate, and Doumas methods were analyzed. The addition of eltrombopag resulted in a concentration-dependent yellowish-brown discoloration of the serum samples and an increase in absorbance from 340 to 640 nm. The lipemia index significantly increased with increasing eltrombopag concentrations. Fourteen patients displayed triglyceride values below 2.60 mmol/L, one of whom exceeding the lipemia index threshold and exhibiting a yellowish-brown serum with an absorbance increase from 340 to 640 nm. The TB measurements showed a statistically significant positive bias across all three methods, with varying degrees of interference: enzymatic (280.2 %), vanadate (124.6 %), and Doumas (323.9 %) at 40.0 µg/mL eltrombopag. Moreover, uric acid levels showed a statistically significant, concentration-dependent negative bias. This study provides valuable insights into eltrombopag interference, offering well-defined methodological details to assist clinical laboratories in accurately interpreting the patient results.

News (Medical) associated with Eltrombopag Diolamine

07 Jul 2025

·www.einpresswire.com

Medicare Cuts to Critical Cancer Care Leave Civil Rights Icon Charles D. Neblett, PhD, Denied Coverage

Black Prostate Check Challenge™ Enters Its Second Year with Ni'Cole Gipson, Founder of NGPR Strategic Communications at the Helm Woke Up This Morning with My Mind Stayed on Freedom”—once a hymn, Neblett helped turn it into a civil rights rally cry. Facing the eve of Independence Day, his verse is medical, not metaphor.” — Charles D. Neblett, PhD. LOS ANGELES, CA, UNITED STATES, July 7, 2025 / EINPresswire.com / -- As the Medicare Prior Authorization Rule—meant to speed up access to treatment through electronic approvals—took effect this July across the U.S., and new Medicaid cuts were signed into law, civil rights icon Dr. Charles D. Neblett of Kentucky is still denied the critical cancer medication his doctors prescribed. The 84-year-old SNCC Freedom Singer, now battling stage 4 metastatic prostate cancer, received approval for Promacta™, a critical platelet-stabilizing medication. But the drug is suddenly not covered due to a complex tangle of shifting policies, coverage classifications, and insurer protocols. Despite meeting medical need, the red tape has pushed his monthly out-of-pocket cost from manageable to unimaginable. In Neblett’s case, that delay may prove fatal. He has gone over two months without Promacta™, and his platelet count has dropped to a dangerously low threshold, raising the risk of internal bleeding, stroke, or trauma-induced death. The drug, which helps his bone marrow produce platelets, remains blocked—even under the new CMS policy intended to speed access to lifesaving treatments. “Woke up this morning with my mind stayed on freedom.” It was a spiritual before it became a rallying cry—but reimagined for protest and mass meetings. Charles Neblett sang it through tear gas and jail bars as a founding member of the SNCC Freedom Singers. July is intended for America to celebrate freedom. But this Freedom Singer sings a different verse—one rooted in medical urgency, not metaphor. “This isn’t freedom,” Neblett says. “Not if I’m fighting cancer while still battling for the medicine that keeps my blood from breaking down.” His concern is echoed by Black Prostate Check Challenge™ panelist Dr. Clayton Yates, a John R. Lewis Professor of Pathology and Director of Translational Health Disparities Research at Johns Hopkins School of Medicine. Dr. Yates affirms: “Black men are more likely to experience low platelet counts during prostate cancer treatment—and if your platelets drop too low, your blood can’t clot. That raises the risk of hemorrhagic stroke.” Johns Hopkins University, where Yates leads multiple initiatives, is a global hub for prostate cancer research and care innovation. It was the birthplace of modern prostate cancer diagnostics and has developed leading surgical and clinical techniques used worldwide today. Its team includes one of the largest and most influential groups of prostate cancer clinicians and researchers, known for their work in urologic oncology, cancer genetics, and health equity. Yates’s clinical findings on Black men and prostate cancer are regularly published and were recently featured in " Nature Communications " demonstrating the link between inflammation-related immune responses and more lethal tumor profiles in Black men. These biological differences, combined with systemic insurance obstacles, help explain why mortality rates remain highest among Black prostate cancer patients. Still, Charles and his family face mounting uncertainty. His prescribed medication remains inaccessible. His condition, though stable, is fragile. And the new CMS rule, celebrated in national headlines, has yet to bring real relief. Marvinia Benton Neblett, Charles’s wife of over 50 years and his full-time caregiver, underscores the urgency. “This campaign is about visibility, yes—but also survival,” she says. “Health equity means nothing if people can’t access the medication their doctors prescribe. What does this say about how we treat our elders—especially in Black communities, where the combination of stroke and cancer is already too common?” Charles Neblett, Ph.D., was honored with the Pioneer of Justice Award by the NAACP Legal Defense Fund (#NEJAD2025), commemorating the 60th anniversary of Selma. The legacy of Dr. Charles D. Neblett inspired the Black Prostate Check Challenge™, a national campaign launched by NíCole Gipson of NGPR Strategic Communications exactly one year ago after Neblett announced his metastatic stage 4 cancer diagnosis. The campaign has now reached over 67 million people, sparking vital conversations across barbershops, newsrooms, and churches—helping shift public awareness and improve shared decision-making in Black men’s healthcare. Now in Year Two, Gipson elevates the campaign—Black Prostate Check Challenge™—expanding beyond digital storytelling across multiple platforms, including the development of: - A children’s book series focused on generational health and a biography of Dr. Neblett’s legacy - A health equity docuseries and feature film—rooted in the real-life struggles and civil rights legacy of Charles - A podcast dedicated to patient voices and systemic change Gipson, founder of NGPR Strategic Communications, is advancing the campaign’s blueprint by developing strategically aligned projects with publishers, podcasters, producers, and funders—while collaborating closely with legacy leaders, journalists, doctors, and grassroots organizers. “My work has always centered on high-profile storytelling—from groundbreaking sitcoms to grassroots civil rights,” Gipson says. “But this campaign expanded me into public service journalism with real stakes. It’s not just storytelling—it’s survival strategy.” As momentum builds, the team is entering a new phase. Doug Davis of the Black Information Network co-created the campaign name and is shaping the next phase of multimedia expansion. “This challenge is about real voices,” Davis says. “We need media that shows what Black families go through while it’s still happening—not after the fact.” This next phase of the campaign will also honor Charles Neblett’s full legacy—from his earliest days marching with John Lewis and being jailed for nonviolent protest, to his current fight navigating the healthcare system as an elder, advocate, and grandfather. “One wrong step, one untreated bleed, and it can be fatal,” Neblett says. “That kind of fragility is hard to speak out loud—I’ve marched through tear gas and buried friends with stronger bodies than mine right now. But my faith and this campaign keep me going.” An urgent national fundraiser is now live to help Neblett access emergency Promacta™ doses and support Black Prostate Check Challenge™. Every delay increases the risk. Every voice helps restore strength. HOW TO HELP: 🔗 https://givebutter.com/CharlesNeblett-CancerFund 📫 Checks payable to: Community Projects, Inc. (501c3) 571 E 7th Street Russellville, KY 42276 📞 Contact: Marvinia Benton Neblett – (270) 957-2836 Note to Editors: This release may be excerpted or reprinted. Credit: Ni'Cole Gipson, NGPR Strategic Communications. Media Relations Department NGPR +1 314-824-8311 nicolemgipson@gmail.com Visit us on social media: LinkedIn Instagram Facebook YouTube TikTok Other Black Prostate Check Challenge Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Drug Approval

02 Jul 2025

·www.einpresswire.com

Civil Rights Icon Charles D. Neblett, PhD, Denied Critical Cancer Care Coverage Despite July 1 Medicare Policy Change

Denied Cancer Treatment Coverage Despite New Medicare Policy, Civil Rights Icon Inspires National Fundraiser as Black Prostate Check Challenge™ Enters Year Two Woke Up This Morning with My Mind Stayed on Freedom”—once a hymn, Neblett helped turn it into a civil rights rally cry. Facing the eve of Independence Day, his verse is medical, not metaphor.” — Charles D. Neblett, PhD., Civil Rights Icon LOS ANGELES, CA, UNITED STATES, July 2, 2025 / EINPresswire.com / -- As the CMS Interoperability and Prior Authorization Final Rule took effect this week—requiring faster insurance decisions and electronic submissions—civil rights icon Dr. Charles D. Neblett is still waiting. The 84-year-old SNCC Freedom Singer, now battling stage 4 metastatic prostate cancer, has yet to receive approval for Promacta™, a critical platelet-stabilizing medication. Despite federal reforms and public pledges by insurers like Humana, Aetna, and UnitedHealthcare, delays persist. In Neblett’s case, those delays may prove fatal. For over two months, Neblett has gone without Promacta™, and his platelet count has dropped to dangerously low threshold of irreversible damage that increases the risk of internal bleeding, hemorrhagic stroke, or trauma-induced death. The medication, which helps his bone marrow produce platelets, remains blocked by insurance even under the new CMS policy meant to prevent such life-threatening delays. Even as Medicare announces steps to improve prior authorization, Neblett warns: a pledge is not a protection. “The same companies delaying my care now signed voluntary pledges back in 2018. If they meant it, I wouldn’t be fighting for this medicine today.” “Woke up this morning with my mind stayed on freedom.” It was a spiritual before it became a rallying cry—but reimagined for protest and mass meetings. Charles Neblett sang it through tear gas and jail bars as a founding member of the SNCC Freedom Singers. Facing the eve of Independence Day, he sings a different verse—one rooted in medical urgency, not metaphor. “This isn’t freedom,” Neblett says. “Not if I’m fighting cancer while still battling for the medicine that keeps my blood from breaking down.” His concern is echoed by Black Prostate Check Challenge panelist™️ Dr. Clayton Yates, a John R. Lewis Professor of Pathology and Director of Translational Health Disparities Research at the Johns Hopkins School of Medicine. Dr. Yates affirms: “Black men are more likely to experience low platelet counts during prostate cancer treatment—and if your platelets drop too low, your blood can’t clot. That raises the risk of hemorrhagic stroke.” Johns Hopkins, where Yates leads multiple initiatives, is a global hub for prostate cancer research and care innovation. It was the birthplace of modern prostate cancer diagnostics and has developed leading surgical and clinical techniques used worldwide today. Its team includes one of the largest and most influential groups of prostate cancer clinicians and researchers, known for their work in urologic oncology, cancer genetics, and health equity. Yates’s clinical findings on Black men and prostate cancer are regularly published and were recently featured in Nature Communications to demonstrate the link between inflammation-related immune responses and more lethal tumor profiles in Black men. These biological differences, combined with systemic insurance obstacles, help explain why mortality rates remain highest among Black prostate cancer patients. Still, Charles and his family face mounting uncertainty. His prescribed medication remains inaccessible. His condition, though stable, is fragile. And the new CMS rule, celebrated in national headlines, has yet to bring real relief. Marvinia Benton Neblett, Charles’s wife of over 50 years and his full-time caregiver, underscores the urgency. “This campaign is about visibility, yes—but also survival,” she says. “Health equity means nothing if people can’t access the medication their doctors prescribe. What does this say about how we treat our elders—especially in Black communities, where the combination of stroke and cancer is already too common?” Charles Neblett, Ph.D. was honored with the Pioneer of Justice Award by the NAACP Legal Defense Fund (#NEJAD2025), commemorating the 60th anniversary of Selma. The legacy of Dr. Charles D. Neblett inspired the Black Prostate Check Challenge™, a national campaign launched by NíCole Gipson of NGPR Strategic Communications exactly one year ago after Neblett announced his metastatic stage 4 cancer diagnosis. The campaign has now reached over 67 million people, sparking vital conversations across barbershops, newsrooms, and churches—helping shift public awareness and improve shared decision-making in Black men’s healthcare. Now in Year Two, Gipson elevates the campaign—Black Prostate Check Challenge™️ into its second year, expanding beyond digital storytelling into structured platforms, including development of: • A children’s book series focused on generational health and a biography of Dr. Neblett’s legacy • A health equity docuseries, feature film — rooted in the real-life struggles and civil rights legacy of Charles • A podcast dedicated to patient voices and systemic change Gipson, founder of NGPR Strategic Communications, continues the blueprint for the campaign strategy in across multiple platforms and in coordination with legacy leaders, journalists, doctors, and grassroots organizers. “My work has always centered on high-profile story telling—from ground breaking sitcoms to grassroots civil rights,” Gipson says. “But this campaign expanded me into public service journalism with real stakes. It’s not just storytelling—it’s survival strategy.” As momentum builds, the team is entering a new phase. Doug Davis of Black Information Network co-created the campaign name and is shaping the next phase of multimedia expansion. “This challenge is about real voices,” Davis says. “We need media that shows what Black families go through while it’s still happening—not after the fact.” This next phase of the campaign will also honor Charles Neblett’s full legacy—from his earliest days marching with John Lewis and being jailed for nonviolent protest, to his current fight navigating the healthcare system as an elder, advocate, and grandfather. “One wrong step, one untreated bleed, and it can be fatal,” Neblett says. “That kind of fragility is hard to speak out loud—I’ve marched through tear gas and buried friends with stronger bodies than mine right now. But my faith and this campaign keep me going.” The urgency remains. A national fundraiser is now live to help Neblett access emergency Promacta™ doses and support BPCC 2.0. Every delay increases the risk. Every voice helps restore strength. HOW TO HELP: 🔗 https://www.givebutter.com/helpcharlesneblett 📫 Checks payable to: Community Projects, Inc. (501c3) 571 E 7th Street Russellville, KY 42276 📞 Contact: Marvinia Benton Neblett – (270) 957-2836 Note to Editors: This release may be excerpted or reprinted with credit to NGPR Strategic Communications and the Black Prostate Check Challenge™. Promacta™ is a trademark of Novartis AG. Media Relations Team NGPR +1 314-824-8311 email us here Visit us on social media: LinkedIn Instagram Facebook YouTube TikTok Other Black Prostate Check Challenge™ Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

12 Jun 2025

·www.sobi.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi’s commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: 1.Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. 2. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 3. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. “The breadth of data that we share at this year’s EHA congress demonstrates Sobi’s comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 DLBCL PS1911: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large BCell Lymphoma (DLBCL) Presenting Author: Juan Pablo Alderuccio Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1957: Updated Safety Run-in Results from LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine with Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL Presenting Author: Carmelo Carlo-Stella Immune Thrombocytopenia (ITP) PF1236: Platelet Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia Who Switched from Eltrombopag or Romiplostim: the REAL-AVA 2.0 Real-World Study Presenting Author: Shruti Chaturvedi Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1239: Durability of Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia: The REAL-AVA 2.0 Real-World Study Presenting Author: Srikanth Nagalla PF1251: Clinically Meaningful Response to Avatrombopag: A Phase 3B Trial for Treatment of Children with ITP Presenting Author: Rachael F. Grace PS2231: Effectiveness and safety of avatrombopag for the treatment of adults with newly diagnosed, persistent, or chronic immune thrombocytopenia: Interim results from the phase 4 ADOPT study Presenting Author: Waleed Ghanima Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a phase 3, randomized, double-blind, placebo-controlled trial Presenting Author: Rachael F. Grace PS2239: Real-World Treatment Patterns & Clinical Outcomes in Thrombopoietin Receptor Agonist Naive Patients with Immune Thrombocytopenia Treated with Avatrombopag: Interim Results from the REAL-AVA 3.0 Study Presenting Author: Sandhya Panch PS2242: Effectiveness and safety of avatrombopag for treatment of immune thrombocytopenia in older patients and those with comorbidities or prior TPO-RA exposure: Interim results from the phase 4 ADOPT study Presenting Author: María Eva Mingot-Castellano PS2244: Response to Avatrombopag Among Patients with Chronic and Persistent Primary Immune Thrombocytopenia: the REAL-AVA 2.0 Real-World Study Presenting Author: M Y Levy PS2250: Evaluation of Efficacy and Safety of Avatrombopag in Children with Immune Thrombocytopenia based on Disease Duration: Results from the Avatrombopag Phase 3-b Pediatric Trial Presenting Author: Rachael F. Grace PB3676: Baseline Correlates with Durability of Avatrombopag Response: A Phase 3B Trial for Treatment of Children with ITP Publication Only Published on May 14 at 15:30 CEST PB3684: Consistent Response to Avatrombopag across Various Baseline Characteristics: Results from the Phase 3-b Trial for the Treatment of Children with Immune Thrombocytopenia Myelofibrosis PF849: Hematologic improvement experienced by pacritinib-treated patients with myelofibrosis in real-world clinical settings Presenting Author: Michael Marrone Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1242: Efficacy of pacritinib vs momelotinib in patients with thrombocytopenic MF: a matched adjusted indicated treatment comparison Presenting Author: Koo Wilson PF1306: Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial Presenting Author: Abiola Oladapo PS1827: Real-world effectiveness of pacritinib in patients with myelofibrosis who have concurrent thrombocytopenia and anemia Presenting Author: Raajit Rampal Poster presentation Session title: Poster Session 2 Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1842: Real-World Treatment Patterns and Clinical Outcomes in Patients with Myelofibrosis Treated with Pacritinib (PAC) with platelets ≥50 x109/L at PAC initiation: Interim results from the MY-PAC Study Presenting Author: Doug Tremblay PS2295: Economic Burden of Cytopenia in Patients with Myelofibrosis: Analysis of a US National Administrative Claims Database Presenting Author: Lucia Marasova PB3079: Cytopenia is associated with real-world disease progression and diminished survival in patients with myelofibrosis: Analysis of a US national administrative claims database Publication Only Published on May 14 at 15:30 CEST Paroxysmal Nocturnal Hemoglobinuria PF672: Early response in complement inhibitor naïve patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan in the Phase 3 PRINCE trial Presenting Author: Austin Kulasekararaj Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF676: Interim analysis of the ongoing COMPLETE study on the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) Presenting Author: Regis Peffault de Latour PS1662: A benefit assessment of pegcetacoplan dose increase in the Phase 3 PEGASUS trial of PNH patients with difficult-to-control disease Presenting Author: Morag Griffin Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS1665: Benefit of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria irrespective of baseline transfusion status Presenting Author: Britta Höchsmann Primary Hemophagocytic Lymphohistiocytosis (pHLH) PF1036: Emapalumab in patients with primary hemophagocytic lymphohistiocytosis: Efficacy and safety outcomes from a multinational, open-label, single-arm study Presenting Author: Franco Locatelli Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine)Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About 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. We provide access to innovative treatments that transform life for people. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.

Phase 3ImmunotherapyClinical ResultDrug ApprovalPhase 1

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.

to view more data

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

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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

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04328727 (REACTS, CTgov)	Phase 2	Anemia, Aplastic \| Refractory Severe Aplastic Anemia	36	rabbit anti-thymocyte globulin+cyclosporine A+Eltrombopag	pgxctxsoff = fqngyivonq chtlnpjwnx (mjhruaijap, njtrpeezxb - zfloloeynd) View more	-	17 Jul 2025
EHA2025 Manual	Not Applicable	Hepatitis \| Anemia, Aplastic	17	Total	yttswgczci(xspkcisvrj) = jtficnlgnl sklviyoweu (cxtxdrtxdh ) View more	Positive	12 Jun 2025
				Avatrombopag	yttswgczci(xspkcisvrj) = olrlsigltg sklviyoweu (cxtxdrtxdh ) View more
EHA2025	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	267	Romiplostim	zcnnsewpkn(ndikaqlhzy) = hlhnahyulp qsgsrktlil (zhrwahsilr ) View more	-	14 May 2025
				Eltrombopag	pkjkezpwaa(qufwmhbqoa) = dofqjlacsy snvkhhnhdx (snjrjlxvxo ) View more
REAL-AVA 2.0 (EHA2025)	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	79	Eltrombopag	ymawratkoz(rlrdvorkvg) = ecmbwatryw lybdsilrmq (edyqdgxhyx ) View more	Positive	14 May 2025
				Romiplostim	ymawratkoz(rlrdvorkvg) = ptdakbizqe lybdsilrmq (edyqdgxhyx ) View more
EHA2025	Not Applicable	Thrombosis	267	Eltrombopag	kkpzckpsqx(axnrdkavxk) = Among the 35 vascular AEs observed in 31 patients, 51% were arterial and 49% were venous, including embolism and deep vein thrombosis fatjetkmkj (fqbcobbfgk )	-	14 May 2025
				Romiplostim
NCT04346654 (XPAG-ITP, CTgov)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	26	Eltrombopag+Dexamethasone (Eltrombopag + Dexamethasone)	xqlekjfrxq = qnchrxjxgt aeuvulsxrf (wornlykjoo, wlpsasvcef - bcfbraaoga) View more	-	17 Mar 2025
				Dexamethasone (Dexamethasone)	xqlekjfrxq = podzknmben aeuvulsxrf (wornlykjoo, ilpnajsyih - xilogajxij) View more
ASH2024 Manual	Phase 2	Purpura, Thrombocytopenic, Idiopathic First line	23	Low-Dose Corticosteroids (LD-CIS) + Low-Dose Eltrombopag (LD-EPAG)	otzzpjaavv(ooyjgrxtgk) = uhkkkercsb bdkvnfwdxr (ppsopimybc ) View more	Positive	09 Dec 2024
NCT03939637 (ASH2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	-	Eltrombopag	cnzpcahrmj(lvhdtcajwc) = 20 AEs grade 3 or higher (including 6 SAEs) in the epag arm, and 6 (3 SAEs) in the SOC arm during the first 12 weeks. The most common AEs were headache (3 epag, 3 SOC) and epistaxis (1 epag, 2 SOC). Drug-related SAEs occurred in 6 pts (epag - 2 elevated LFTs, 2 headache (HA); SOC - 1 allergic reaction, 1 HA). There were no thromboembolic events. There was 1 intracranial hemorrhage in the epag arm. zsbmevzcvp (gyrkofgzyy )	-	09 Dec 2024
				Standard First-Line Therapy (SOC)
RACE trial (ASH2024)	Phase 3	Anemia, Aplastic First line	-	Eltrombopag plus IST	fjrteedvdq(bhppzifhre) = juqphxbcgc ntxhcuivle (dupelrmmyk )	Negative	08 Dec 2024
				IST alone	fjrteedvdq(bhppzifhre) = izrklyqgun ntxhcuivle (dupelrmmyk )
ASH2024	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	-	Eltrombopag	ogunnyevky(fjojjwjzkq) = rgknczpyxx kpwyclcqmo (faoduiozex ) View more	-	08 Dec 2024
				Romiplostim	ogunnyevky(fjojjwjzkq) = atgyyviken kpwyclcqmo (faoduiozex ) View more

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