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Last update 08 Jul 2025

Avatrombopag Maleate

Last update 08 Jul 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

avatrombopag, Avatrombopag maleate (JAN/USAN), AKR-501

+ [14]

Target

TPO receptor

Action

agonists

Mechanism

TPO receptor agonists(Thrombopoietin receptor agonists)

Therapeutic Areas

Immune System DiseasesDigestive System DisordersHemic and Lymphatic Diseases+ [2]

Active Indication

Chronic liver diseasePurpura, Thrombocytopenic, IdiopathicThrombocytopenia+ [3]

Inactive Indication

Alpha 1-Antitrypsin DeficiencyCardiovascular DiseasesChronic idiopathic thrombocytopenic purpura+ [14]

Originator Organization

Astellas Pharma, Inc.

Active Organization

Sobi North America

Swedish Orphan Biovitrum AB

Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd.

+ [4]

Inactive Organization

Eisai Co., Ltd.

Eisai, Inc.

Dova Pharmaceuticals, Inc.

+ [2]

License Organization

Shanghai Fosun Pharmaceutical (Group) Co., Ltd.

Dova Pharmaceuticals, Inc.

Swedish Orphan Biovitrum Ltd.

+ [6]

Drug Highest PhaseApproved

First Approval Date

United States (21 May 2018),

Thrombocytopenia

RegulationOrphan Drug (United States), Priority Review (China)

Structure/Sequence

Molecular FormulaC33H38Cl2N6O7S2

InChIKeyMISPBGHDNZYFNM-BTJKTKAUSA-N

CAS Registry677007-74-8

103

Clinical Trials associated with Avatrombopag Maleate

NCT07051915

/ Not yet recruitingPhase 3IIT

Comprehensive Evaluation of Two Second-Line Therapeutic Approaches for Immune Thrombocytopenia (ITP) - a Pragmatic Randomized Controlled Trial

This study is a phase 3 study where eligible patients will be randomized 1:1 to one of two treatment strategies: receiving a thrombopoietin receptor agonist (Avatrombopag), or anti-CD20 (Rituximab).

Start Date01 Jul 2025

Sponsor / Collaborator

Nordlandssykehuset HF

[+10]

CTR20251305

/ CompletedNot Applicable

马来酸阿伐曲泊帕片在中国健康参与者中的随机、开放、两制剂、两序列、四周期、完全重复交叉、餐后状态下生物等效性试验

[Translation] A randomized, open-label, two-dose, two-sequence, four-period, completely repeated crossover, fed-state bioequivalence study of avatrombopag maleate tablets in healthy Chinese participants

主要目的：以江苏奥赛康药业有限公司生产的马来酸阿伐曲泊帕片（规格：20 mg）为受试制剂，按生物等效性试验的有关规定，与AKaRx Inc.持证、Kawashima Plant, Eisai Co.,Ltd.生产的马来酸阿伐曲泊帕片（规格：20 mg，商品名：苏可欣®，参比制剂）对比在健康人体内的相对生物利用度，考察两制剂的餐后状态下生物等效性。次要目的：观察马来酸阿伐曲泊帕片受试制剂和参比制剂（商品名：苏可欣®）在健康参与者中餐后状态下给药的安全性。

[Translation]

Primary objective: To compare the relative bioavailability of Avatrombopag Maleate Tablets (specification: 20 mg) produced by Jiangsu Aosaikang Pharmaceutical Co., Ltd. with Avatrombopag Maleate Tablets (specification: 20 mg, trade name: Sukoxin®, reference preparation) produced by AKaRx Inc. and Kawashima Plant, Eisai Co., Ltd. in healthy human bodies according to the relevant provisions of bioequivalence test, and to investigate the bioequivalence of the two preparations in the postprandial state. Secondary objective: To observe the safety of the Avatrombopag Maleate Tablets test preparation and the reference preparation (trade name: Sukoxin®) in the postprandial state in healthy participants.

Start Date07 May 2025

Sponsor / Collaborator

Jiangsu Aosaikang Pharmaceutical Co. Ltd.

CTRI/2024/08/072877

/ CompletedNot Applicable

An Open Label Balanced Randomized Single Dose Two Treatment Three Sequence Three Period Partial Replicate Reference Scaled Average Oral Bioequivalence Study of Avatrombopag Maleate 20 Mg Tablets Manufactured by Bdr Pharmaceuticals Internationals Pvt Ltd India with Doptelet (avatrombopag) 20 mg Tablets Manufactured By Swedish Orphan Biovitrum Ab Publ Stockholm Sweden Se 11276 In Healthy Adult Human Subjects Under Fed Conditions - Nil

Start Date04 Sep 2024

Sponsor / Collaborator

BDR Pharmaceuticals International Pvt Ltd.

100 Clinical Results associated with Avatrombopag Maleate

100 Translational Medicine associated with Avatrombopag Maleate

100 Patents (Medical) associated with Avatrombopag Maleate

241

Literatures (Medical) associated with Avatrombopag Maleate

01 Jul 2025·Lancet Haematology

Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study

Article

Author: Yesilipek, Mehmet Akif ; Sandham, Jessica ; Fritch-Lilla, Stephanie A ; Aydinok, Yesim ; Motwani, Jayashree ; Laguna, Pawel ; Connor, Philip ; Dudnyk, Veronika ; Plaksina, Olga ; Astwood, Emma J ; Alhushki, Waseem ; Sibson, Keith ; Zhang, Jessica ; Kurylak, Andrzej ; Leblebisatan, Göksel ; Mlynarski, Wojciech ; Grainger, John ; Holzhauer, Susanne ; Schultz, Corinna L ; Hauser, Peter ; Yakarisik, Selin ; Unal, Selma ; Kovacs, Gabor ; Jamieson, Brian D ; Grace, Rachael F ; de León, Emily ; Donyush, Elena ; Ünal, Sule ; Karakas, Zeynep ; Smallwood, Linda ; Rose, Melissa J ; Kupesiz, Osman Alphan ; Grainger, John D ; Hsieh, Loan B ; Pasquet, Marlene ; Grimes, Amanda B ; Popova, Nataliia ; Ottoffy, Gabor ; Strahm, Brigitte ; Ünal, Şule

BACKGROUND:

Avatrombopag is an oral thrombopoietin receptor agonist (TPO-RA), taken with food without any specific restrictions, approved for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The aim of this study was to assess the efficacy, safety, tolerability, and pharmacokinetic and pharmacodynamic profile of avatrombopag for children and adolescents with persistent and chronic primary ITP.

METHODS:

AVA-PED-301 is a global, multicentre, randomised, double-blind, placebo-controlled, parallel-group, phase 3b study. Children and adolescents aged ≥1 to <18 years with a confirmed diagnosis of primary ITP for at least 6 months and an insufficient response to a previous treatment were randomly assigned (3:1) to avatrombopag (aged ≥6 years: 20 mg oral tablet once daily; aged ≥1 to <6 years: 10 mg oral suspension once daily) or matching placebo. Doses were titrated to maintain a platelet count of 50-150 × 10⁹ cells per L. The core-phase (12-week double-blind treatment period) full analysis set included all randomly assigned patients; efficacy analysis was performed based on randomised study drug. The safety analysis set included all patients who received at least one dose of study drug in each study phase. The primary endpoint was durable platelet response (proportion of patients with at least six out of eight weekly platelet counts ≥50 × 10⁹ cells per L during the last 8 weeks of the 12-week treatment period in the absence of rescue therapy); the alternative primary endpoint was platelet response (proportion of patients with at least two consecutive platelet assessments ≥50 × 10⁹ cells per L over the 12-week treatment period in the absence of rescue therapy). The trial core phase is complete and was registered with ClinicalTrials.gov (NCT04516967).

FINDINGS:

Between March 2, 2021, and Aug 2, 2023, 83 children were screened, with 75 randomly assigned to avatrombopag (n=54; 24 [44%] female; 48 [89%] White) or placebo (n=21; 12 [57%] female; 15 [71%] White). 15 (28%) patients in the avatrombopag group met the primary endpoint of durable platelet response versus no (0%) patients in the placebo group (difference in response rate 28% [95% CI 16-40]; p=0·0077); 44 (81%) patients in the avatrombopag group met the alternative primary endpoint of platelet response versus no (0%) patients in the placebo group (difference in response rate 81% [71-92]; p<0·0001). The most common adverse events across treatment groups were petechiae (n=20), epistaxis (n=16), and headache (n=14). Serious adverse events were reported in five (9%) patients in the avatrombopag group and one (5%) patient in the placebo group. No deaths, thromboembolic events, or grade 3 or higher bleeding events were reported.

INTERPRETATION:

Avatrombopag is an effective oral treatment for children and adolescents with ITP for at least 6 months and has a reassuring safety profile in the paediatric population. Avatrombopag could provide an important treatment option for paediatric ITP.

FUNDING:

Sobi.

01 Jul 2025·HEPATOLOGY RESEARCH

Autoimmune hepatitis following transition from prednisolone to thrombopoietin receptor agonist therapy in immune thrombocytopenic purpura: A clinical precaution

Article

Author: Kakisaka, Keisuke ; Miyasaka, Akio ; Suzuki, Akiko ; Fujiwara, Yudai ; Kuroda, Hidekatsu ; Abe, Tamami ; Endo, Kei ; Sasaki, Tokio ; Inarimori, Kaname ; Yoshida, Yuichi ; Watanabe, Takuya ; Matsumoto, Takayuki

Abstract:

Background:

Autoimmune hepatitis (AIH) occasionally complicates immune thrombocytopenic purpura (ITP). The risk of AIH development after prednisolone (PSL) discontinuation and thrombopoietin receptor agonist (TPO‐RA) initiation remains underrecognized.

Aim and Methods:

We report two cases of AIH that developed or exacerbated after switching from PSL to TPO‐RAs (avatrombopag and eltrombopag) for ITP treatment.

Results:

Case 1 developed AIH 8 months after PSL discontinuation and avatrombopag initiation. Case 2 experienced AIH exacerbation with acute decompensation of liver cirrhosis 2 months after PSL discontinuation while on eltrombopag. Both cases showed no improvement after TPO‐RA discontinuation but responded well to PSL reintroduction.

Conclusions:

PSL discontinuation likely unmasks underlying AIH in ITP patients, with TPO‐RAs potentially modulating immune responses. Regular liver function monitoring is essential when transitioning ITP patients from PSL to TPO‐RAs.

10 Jun 2025·Blood Advances

Safety, efficacy, and treatment satisfaction in adults with ITP who switched to avatrombopag from another TPO-RA

Article

Author: Zhang, Jessica ; Jamieson, Brian D. ; Kolodny, Scott ; Tarantino, Michael D. ; Mosalpuria, Kailash ; Vredenburg, Michael

Abstract:

This phase 4, multicenter, open-label study was conducted to evaluate the safety, efficacy, and treatment satisfaction of switching to avatrombopag from another thrombopoietin receptor agonist (TPO-RA) in patients with immune thrombocytopenia (ITP). Adults who had received ≥90 days of treatment with eltrombopag or romiplostim and had a response (2 platelet counts [PCs] ≥50 × 109/L) switched to avatrombopag with no protocol-defined washout period. The primary end point was the incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs. Secondary end points were the proportion of patients who had a PC between ≥50 × 109/L and ≤200 × 109/L (days 15, 30, 60, and 90) and change from baseline in each domain of the self-administered Treatment Satisfaction Questionnaire for Medication (TSQM) to day 90. Among 60 enrolled patients, 58.3% experienced TEAEs and 10.0% experienced serious TEAEs (1 related to avatrombopag [thrombocytopenia that resolved]; 5 unrelated [1 unrelated death]). A PC ≥50 × 109/L to ≤200 × 109/L was reported for 51.7%, 31.7% (mean PC, 256.2 × 109/L [standard deviation, 176.7 × 109/L]), 55.0%, 60.0%, and 55.0% at baseline and on days 15, 30, 60, and 90, respectively. TSQM scores increased from baseline to day 90 across all domains (mean change: convenience, +13.5; effectiveness, +14.4; global satisfaction, +14.2; side effects, +8.3). There was no correlation between stable avatrombopag dose (day 90) and previous TPO-RA dose (high or low). Patients with ITP may safely switch from another TPO-RA to avatrombopag and maintain adequate PCs while experiencing improved treatment satisfaction. This trial was registered at www.ClinicalTrials.gov as #NCT04638829

News (Medical) associated with Avatrombopag Maleate

20 Jun 2025

·www.prnewswire.com

Sobi will showcase extensive research and clinical outcomes at ISTH 2025

STOCKHOLM, June 20, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) will be presenting a wide range of clinical study updates at the annual International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington (21-25 June). With ten (10) presentations, Sobi's clinical teams will showcase new data for avatrombopag to treat children with ITP, surgery outcomes using efanesoctocog alfa, and abstracts on specific long-term haemophilia studies. "Attending ISTH is an important part of our commitment to collaboration and knowledge sharing so we can further advance treatments for those living with haemophilia, immune thrombocytopenia, and other rare blood conditions", said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. "We are proud to be showcasing abstracts as part of the ISTH programme, including Doptelet abstracts that present new data related to children living with ITP, Altuvoct outcomes for perioperative management, and joint health outcomes from a long-term extension study, as well as final outcomes from the B-MORE study. Many people living with rare conditions have unmet needs, and we are proud to present additional data advancing treatments for these rare conditions." Data to be presented at ISTH 2025 All abstracts are accessible through the official ISTH website. However, any late-breaking abstracts will only be made available later. About ALTUVOCT® (efanesoctocog alfa) ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A. ALTUVOCT can be used for all age groups and any disease severity. About Elocta®/Eloctate® (efmoroctocog alfa) Elocta®/Eloctate® (efmoroctocog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia A. Elocta/Eloctate can be used for all age groups. About Alprolix® (eftrenonacog alfa) Alprolix® (eftrenonacog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia B. Alprolix can be used for all age groups. 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 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. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and delivering compelling growth. Sanofi apply deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Sanofi's team is guided by one purpose: to chase the miracles of science to improve people's lives; this inspires Sanofi to drive progress and deliver positive impact for Sanofi's people and the communities Sanofi serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN andNASDAQ: SNY About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix and Elocta/Eloctate. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, or ALTUVIIIO ® in the US, Japan, and Taiwan, and Altuvoct in Europe. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

12 Jun 2025

·www.prnewswire.com

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

STOCKHOLM, June 12, 2025 /PRNewswire/ -- 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: 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. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 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 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. This information was brought to you by Cision . The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyDrug Approval

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. 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Phase 3ImmunotherapyClinical ResultDrug ApprovalPhase 1

100 Deals associated with Avatrombopag Maleate

External Link

KEGG	Wiki	ATC	Drug Bank
D10307	Avatrombopag Maleate	B02BX	DB11995

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Chronic liver disease	Canada	Swedish Orphan Biovitrum AB	03 Nov 2023
Purpura, Thrombocytopenic, Idiopathic	European Union	Swedish Orphan Biovitrum AB	20 Jun 2019
Purpura, Thrombocytopenic, Idiopathic	Iceland	Swedish Orphan Biovitrum AB	20 Jun 2019
Purpura, Thrombocytopenic, Idiopathic	Liechtenstein	Swedish Orphan Biovitrum AB	20 Jun 2019
Purpura, Thrombocytopenic, Idiopathic	Norway	Swedish Orphan Biovitrum AB	20 Jun 2019
Thrombocytopenia	United States	AkaRx, Inc.	21 May 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Primary thrombocytopenia	Phase 3	China	AkaRx, Inc.	02 Mar 2021
Primary thrombocytopenia	Phase 3	China	Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd.	02 Mar 2021
Primary thrombocytopenia	Phase 3	China	Kawashima Group Co., Ltd.	02 Mar 2021
Chronic idiopathic thrombocytopenic purpura	Phase 3	United States	Eisai, Inc.	26 Mar 2012
Chronic thrombocytopenia	Phase 3	Australia	Eisai, Inc.	16 Feb 2012
Chronic thrombocytopenia	Phase 3	Belgium	Eisai, Inc.	16 Feb 2012
Chronic thrombocytopenia	Phase 3	Bulgaria	Eisai, Inc.	16 Feb 2012
Chronic thrombocytopenia	Phase 3	Czechia	Eisai, Inc.	16 Feb 2012
Chronic thrombocytopenia	Phase 3	Netherlands	Eisai, Inc.	16 Feb 2012
Chronic thrombocytopenia	Phase 3	New Zealand	Eisai, Inc.	16 Feb 2012

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04516967 (AVA-PED-301, Pubmed \| Lancet Haematol)	Phase 3	Purpura, Thrombocytopenic, Idiopathic Maintenance	75	Avatrombopag	rtbdgmbkci(dhniemokvj) = yjxpanyhxs frlfjkouih (goyqsgtaho, 16 - 40) View more	Positive	01 Jul 2025
				Placebo	-
EHA2025 Manual	Not Applicable	Anemia, Aplastic	17	Total	szaqwxlarh(qspvqlmftj) = xslzmbbsaj rcilmsmdxf (imqqgcrszk ) View more	Positive	12 Jun 2025
				Avatrombopag	szaqwxlarh(qspvqlmftj) = woocqjterm rcilmsmdxf (imqqgcrszk ) View more
NCT04516967 (Phase 3B trial, EHA2025)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	75	Avatrombopag	xfrrdjjghs(qjxieokgko) = mejemxopsf dgsroxbvah (uxuhuyjxyn )	Positive	14 May 2025
REAL-AVA 3.0 (EHA2025)	Not Applicable	Purpura, Thrombocytopenic, Idiopathic First line	147	Avatrombopag	ytlgjirphf(xymmrmyher) = dmkubajajy xsclwkviia (smuwnivviu ) View more	-	14 May 2025
NCT04516967 (AVA Phase 3-B Pediatric Trial, EHA2025)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	75	Avatrombopag	slbdanyway(yxhvgtwewl) = wsypvavrrd humucezyli (zhzcwhyxmz ) View more	Positive	14 May 2025
				Placebo	slbdanyway(yxhvgtwewl) = jeoikxbnqc humucezyli (zhzcwhyxmz ) View more
ACTRN12619001042134 (DIAAMOND-AVA-NEXT, EHA2025)	Phase 2	Refractory Severe Aplastic Anemia	23	Avatrombopag	qkqmeuicoo(muvodedklq) = uhgvjuxlla rtroasfpsn (xoatirfwqz, 0 - 18) View more	Positive	14 May 2025
REAL-AVA 2.0 (EHA2025)	Not Applicable	Purpura, Thrombocytopenic, Idiopathic Maintenance	171	AVATROMBOPAG (Acute/Persistent ITP)	skpznjwwec(cyksakmmbr) = inhicnuxcw epstoaxqbf (zlmjfhsacj ) View more	-	14 May 2025
				AVATROMBOPAG (Chronic ITP)	skpznjwwec(cyksakmmbr) = hroguurwcp epstoaxqbf (zlmjfhsacj ) View more
REAL-AVA 2.0 (EHA2025)	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	79	Eltrombopag	kfuobjkdgj(tkzztyqnqd) = qrwgebmrbc rggxneeimj (idwtoqyzow ) View more	Positive	14 May 2025
				Romiplostim	kfuobjkdgj(tkzztyqnqd) = qkmvypoaap rggxneeimj (idwtoqyzow ) View more
ADOPT (EHA2025)	Phase 4	Maintenance	200	AVATROMBOPAG (Newly Diagnosed ITP)	qofyquxpme(cvzpcjhlrp) = opbumvrsql gkegfiksiq (knnsxdbejf )	Positive	14 May 2025
				AVATROMBOPAG (Persistent ITP)	qofyquxpme(cvzpcjhlrp) = tvhrntnwau gkegfiksiq (knnsxdbejf )
REAL-AVA 2.0 (EHA2025)	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	177	AVA ≥30k/µL	jiczxurcga(bzxztzwwum) = cchlhdyhsw prhxwjttig (agngipphkk, 5.5 - 23.0)	-	14 May 2025
				AVA ≥50k/µL	prbwtteanu(oisjyaicyi) = ndyapjpvzy pfocvwszqe (gadgpcorwm, 5.3 - 22.4)

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