Delving into the Latest Updates on Eltrombopag Diolamine with Synapse

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

Login to view timeline

Structure/Sequence

Molecular FormulaC29H36N6O6

InChIKeyPLILLUUXAVKBPY-SBIAVEDLSA-N

CAS Registry496775-62-3

The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids.

Start Date08 Feb 2027

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT07687134

/ Not yet recruitingPhase 2IIT

Eltrombopag Combined With Telitacicept Versus Eltrombopag Monotherapy for the Treatment of Immune Thrombocytopenia Refractory or Relapsed to Corticosteroid Therapy: A Randomized Exploratory, Controlled, Open-label, Phase II Clinical Study

Background: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Corticosteroids are first-line therapy, but about one-third of patients relapse or are refractory. Eltrombopag (a TPO-RA) promotes platelet production, yet some patients show no response or relapse upon discontinuation. Telitacicept, a TACI-Fc fusion protein, dual-targets BAFF and APRIL, inhibiting B cell and plasma cell function and reducing autoantibody production, potentially providing synergistic immunomodulatory benefit.
Objective: To evaluate the sustained response rate of eltrombopag plus telitacicept vs. eltrombopag alone in patients with steroid-refractory/relapsed ITP.
Design: Randomized, open-label, controlled, exploratory Phase II study. 40 patients planned (20 combination, 20 monotherapy). Combination group: eltrombopag + telitacicept . Monotherapy group: eltrombopag alone for 12 weeks. Monotherapy patients with no response after 4 weeks may cross over to the combination group. After 12 weeks, treatment is stopped and patients are followed until Week 24.
Primary endpoint: Proportion of patients maintaining platelet count ≥30×10⁹/L with no bleeding at 24 weeks post-treatment. Secondary endpoints include platelet response rates during 12 weeks, safety, bleeding events, etc.
Expected results: The combination group is expected to have a significantly higher proportion of patients achieving the primary endpoint without increased adverse events.
Population: Age ≥18, diagnosed ITP ≥3 months, baseline platelets <30×10⁹/L, prior corticosteroid failure.
Safety: Monitoring for bleeding, infection, thrombosis, cytopenia, etc., with dose adjustment/cessation as per protocol.
Conclusion: This study explores whether dual-targeting (platelet production + autoimmune suppression) with eltrombopag and telitacicept can provide more durable remission for steroid-refractory/relapsed ITP patients.

Start Date10 Jul 2026

Sponsor / Collaborator-

NCT07788456

/ RecruitingPhase 4IIT

Efficacy and Safety of Multi-Target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia: A Prospective, Single-arm, Multicenter, Exploratory Trial

This prospective, single-arm, multicenter, exploratory study will enroll 38 adults patients with newly diagnosed primary immune thrombocytopenia (ITP). Patients will receive standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) as initial therapy During the core treatment phase (Weeks 1-12). Corticosteroids will be tapered and discontinued within 8 weeks, whereas TPO-RA treatment will continue for 12 weeks. For patients with treatment failure( defined as platelet count < 30 × 10^9/L or less than 2-fold increase of baseline platelet count or bleeding) ,a sequential multitarget combination strategy will be explored in subsequent treatment phases. Specifically, patients with treatment failure after 2 weeks of initial therapy (Weeks 3-12), will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb） as sequential combination therapy.
During the core treatment follow-up phase (24 weeks), patients with treatment failure will enter the exploratory treatment phase (Weeks 13-36) .
Patients who received multi-target drug therapy during the core treatment period will switch to an alternative TPO-RA with cross-administered rituximab and anti-CD38 mAb, while those who did not will receive sequential rituximab or anti-CD38 mAb.
Finally, patients will enter the safety follow up period (4 weeks, weeks 37-40).

Start Date22 Jun 2026

Sponsor / Collaborator

Hematology Hospital of Chinese Academy of Medical Sciences

100 Clinical Results associated with Eltrombopag Diolamine

Login to view more data

100 Translational Medicine associated with Eltrombopag Diolamine

Login to view more data

100 Patents (Medical) associated with Eltrombopag Diolamine

Login to view more data

1,479

Literatures (Medical) associated with Eltrombopag Diolamine

31 Dec 2026·Hematology

Efficacy and safety of TPO-RA combined with sirolimus in the treatment of relapsed immune thrombocytopenia

Article

Author: Chen, Xiaoli ; Wang, Dan ; Feng, Yimei ; Zhang, Xi ; Liu, Jia ; Li, Jinqiong ; Tan, Aoxue

BACKGROUND:

Relapsed immune thrombocytopenia (ITP) poses challenges in treatment. Thrombopoietin receptor agonists (TPO-RAs) are recommended as second-line therapy, but the efficacy of monotherapy is limited to about 60%. Our preliminary studies suggest sirolimus is effective for relapsed ITP. This study aimed to evaluate the efficacy and safety of TPO-RA combined with sirolimus in treating relapsed ITP.

METHODS:

A retrospective analysis was conducted on 50 patients with relapsed ITP, all of whom received TPO-RAs (hetrombopag and eltrombopag) combined with sirolimus therapy. The primary endpoints were the overall response rate (ORR) at 4, 8, and 12 weeks of treatment. Secondary endpoints included safety of this regimen and subgroup analysis.

RESULTS:

ORR at 4, 8, and 12 weeks was 61%, 76%, and 82%, respectively, with a median time to response of 7 days. Mean platelet counts at 2-, 4-, 8-, and 12-weeks post-treatment were 71 ± 12.7 × 10⁹/L, 86 ± 10.9 × 10⁹/L, 128 ± 19.7 × 10⁹/L and 131 ± 17.2 × 10⁹/L, respectively. Whether it is hetrombopag or eltrombopag, when combined with sirolimus, there is no statistically significant difference in efficacy between the two subgroups. TPORAs combined with sirolimus as second-line treatment and as third-line or beyond treatment showed no difference in efficacy. Among ANA-positive patients, the ORR shows no significant difference compared to the ANA-negative group (p > 0.05). Patients with ITP for less than one year are more likely to benefit from this treatment regimen (p = 0.003).

CONCLUSIONS:

The combination of TPO-RAs and sirolimus significantly improved platelet counts and sustained response rates. The regimen demonstrated a manageable safety profile, offering a novel therapeutic option for relapsed ITP.

31 Dec 2026·PLATELETS

Single-cell transcriptome deciphers key targets of thrombopoietin receptor agonists and immune microenvironment characteristics of immune thrombocytopenia

Article

Author: Wan, Xiaohan ; Hou, Ming ; Wang, Haoxu ; Wang, Wanru ; Wang, Ruixue ; Cao, Junying

Thrombopoietin receptor agonists (TPO-RAs) represent a cornerstone in immune thrombocytopenia (ITP) management, yet their molecular mechanisms remain incompletely elucidated. This study systematically deciphered the key targets and signaling networks of four TPO-RAs (romiplostim, eltrombopag, avatrombopag, hetrombopag) in ITP pathogenesis. Network pharmacology was integrated with single-cell high-dimensional weighted gene co-expression network analysis (hdWGCNA) using bone marrow scRNA-seq data from ITP patients and healthy controls. Metacell-based co-expression modules to hematopoietic bone marrow cells were identified. Drug targets were curated from multiple databases, and candidate genes were screened by intersecting differentially expressed genes (DEGs), cell specific modules, and TPO-RA targets. Molecular docking, pseudotime trajectory analysis, and in silico gene knockdown were employed for functional validation. Intersection analysis revealed five key genes (CACNA1A, CSF1R, PKN1, CD9, DSTYK). Molecular docking demonstrated strong binding affinities between TPO-RAs and key targets. The ITP bone marrow niche exhibited rewired cell-cell communication, with enhanced T cell-initiated signaling and aberrant megakaryocyte-T cell interactions. Pseudotime analysis uncovered disrupted megakaryocyte maturation dynamics. In silico knockdown revealed CACNA1A, CSF1R, and PKN1 dysregulation exacerbated neutrophil hyperactivity, while CD9 and DSTYK knockdown impaired mitotic regulation. This study delineated mechanisms of TPO-RAs, highlighting five key genes that orchestrate dysregulated thrombopoiesis and immune dysfunction in ITP. The integration of in silico strategies identified novel targets for optimizing ITP therapy.

31 Dec 2026·PLATELETS

Durable platelet response and immature platelet indices following sequential efgartigimod and eltrombopag therapy in a patient with refractory ITP

Article

Author: Takaori-Kondo, Akifumi ; Wada, Fumiya ; Umehara, Hisanori ; Shinkawa, Yutaka ; Nakamura, Takuji ; Satoh-Nakamura, Tomomi ; Matsui, Hiroyuki

Efgartigimod, a neonatal Fc receptor (FcRn) antagonist, represents a novel therapeutic approach for refractory immune thrombocytopenia (ITP). However, its clinical positioning and durability of response in real-world settings remain unclear. We describe a case of refractory ITP in which efgartigimod induced a partial response that was enhanced by the subsequent addition of a thrombopoietin receptor agonist (TPO-RA), resulting in durable remission after efgartigimod discontinuation. Subsequent hematologic workup revealed that platelet recovery was accompanied by a reciprocal shift in immature platelet indices, with immature platelet fraction percentage decreasing and absolute immature platelet fraction increasing. Although there is a paucity of literature on immature platelet indices during ITP treatment, these parameters may serve as useful biomarkers for monitoring therapeutic response. Notably, efgartigimod was discontinued after 22 weeks, with sustained response maintained for an additional 24 weeks on TPO-RA monotherapy. This treatment course highlights the potential of FcRn inhibition to induce durable immunomodulatory effects and demonstrates synergistic benefits with TPO-RAs-insights that extend beyond the scope of conventional clinical trial protocols.

48

News (Medical) associated with Eltrombopag Diolamine

21 Jul 2026

·www.pharmaceutical-technology.com

Novartis Q2 profits recover as revenue mantle passes to newer medicines

Novartis is undergoing one of the most significant portfolio challenges in its history, with loss of exclusivities affecting several of its key assets. Credit: Michael Derrer Fuchs/Shutterstock.com. Despite a lacklustre start to the year, Novartis seems to have overcome its financial slump, reporting operating profits in Q2 that exceeded analyst expectations. The Swiss drugmaker’s core operating income for the quarter was $5.94bn, representing flat year-over-year growth. However, this was ahead of analyst expectations of $5.31bn via Visible Alpha. Quarterly sales grew 1% at constant currency to $14.1bn, which was a 3% beat on expectations as per a research note from Citi analysts. Novartis is undergoing one of the most significant portfolio challenges in its history, with loss of exclusivities affecting several of its key assets. The most important in this group is blockbuster heart failure medication Entresto (sacubitril/valsartan). The drug racked up $7.8bn in peak sales in 2025, though revenue has been plummeting since the core US patent expired last year. That led to a weak Q1 for the company , with profits down 12%. Entresto sales dropped 42% in Q1, and the story has continued into Q2 with the medicine’s sales declining 50%. Other drugs are also suffering from generics market entry, including blood disorder medicine Promacta (eltrombopag) and blood cancer therapy Tasigna (nilotinib). However, in positive momentum for Novartis, new medicines growth meant the drugmaker has stemmed the tide in Q2. Breast cancer drug Kisqali (ribociclib) continued to do much of the heavy lifting, growing 43% compared to the same quarter last year to $1.7bn. Kesimpta (ofatumumab), a multiple sclerosis drug, generated $1.4bn, an increase of 32%. Oncology assets Scemblix (asciminib) and Pluvicto also contributed as growth drivers, alongside cholesterol-lowering injection Leqvio (inclisiran). Scemblix was the highest-growth medication amongst Novartis’ priority brands, up 89% from last year’s same quarter due to strong momentum among newly diagnosed chronic myeloid leukaemia patients. Looking ahead, Novartis reaffirmed its 2026 guidance of low single-digit growth for net sales. Core operating income is expected to decline low single-digit. The company has key trial readouts and new launches to lean on. Chief among these is Bruton’s tyrosine kinase (BTK) inhibitor Rhapsido (remibrutinib). The drug has been racking up regulatory wins over the past year in chronic spontaneous urticaria (CSU), a significant market opportunity. Citi analysts already highlighted a “solid CSU launch, with strong early US uptake” for the drug, which is also approved for the skin disorder in Europe. Novartis CEO Vas Narasimhan said: “Novartis delivered a solid second quarter, returning to sales growth driven by continued momentum from Kisqali, Kesimpta, Scemblix and Pluvicto. We are encouraged by the early trajectory of our recent launches, Rhapsido in CSU and Itvisma. We also made meaningful pipeline progress, highlighted by updated Kisqali overall survival data in early breast cancer and the FDA accelerated approval submission for del-zota in DMD. We are on track for multiple important readouts ahead in the second half and remain on track to deliver our full-year guidance and mid-term outlook.”

Drug ApprovalAccelerated Approval

07 Jul 2026

·www.fiercepharma.com

Novartis dismisses 322 more staffers based out of US headquarters

Novartis is executing more layoffs at its U.S. headquarters in East Hanover, New Jersey, handing 322 employees pink slips. The cuts will go into effect by Oct. 2, according to a Worker Adjustment and Retraining Notification (WARN) Act filing with the Garden State. Novartis is making changes to its field sales, patient support and marketing organizations to “enhance operational efficiency and ensure we are investing resources where they can have the greatest impact," a company spokesperson wrote in an emailed statement. "As a result, some roles are impacted." “Novartis continually evaluates opportunities to align our organization with evolving patient, customer and business needs,” the spokesperson added. It is the fourth round of dismissals over the last five months at the East Hanover site. The other three rounds of cuts, which also were listed in WARN reports, will cost a total of 250 employees their jobs. In March, Novartis moved to eliminate 114 roles at its U.S. home base and explained that it was reworking its sales team for rare disease medicines, including chronic myeloid leukemia treatment Tasigna and platelet-stimulating agent Promacta, both of which are losing patent protection this year. Two months later in May, Novartis said it would chop another 76 positions, with the company citing a downsizing of its biomedical research arm. A few weeks after that, the company telegraphed an additional 60 job cuts, saying that it had “taken steps to evolve our Oncology, Rheumatology, Dermatology and Neuroscience field sales teams in the U.S." Novartis is feeling the pinch of the loss of exclusivity (LOE) of megablockbuster heart failure medicine Entresto, which began in July of last year. Revenue from the heart failure medicine plummeted from $2.3 billion in the first quarter of 2025 to $1.3 billion in this year’s first quarter. With the dropoff, Novartis saw its overall sales decline in the first quarter by 1%, this coming after the company reported revenue increases of 12% in 2024 and 8% in 2025. In March of 2025, Novartis also slashed 427 jobs, citing the upcoming LOE of Entresto. Novartis has been working to adjust and right-size its operations since 2022, when it merged its oncology and pharmaceuticals units into one innovative medicines business, now managed by geographic regions.

01 Jun 2026

·www.fiercebiotech.com

Agios signs $165M deal for blood disorder drug from Korea’s Oscotec that flunked phase 2 study

Oscotec’s phase 2 trial missed its primary goal of demonstrating that cevidoplenib could double a patient’s platelet count after 12 weeks of treatment.\n Days after its lead clinical-stage drug suffered a setback, Agios Pharmaceuticals has rebounded by buying a blood disorder drug that flunked a phase 2 study.The prize is an oral spleen tyrosine kinase (SYK) inhibitor called cevidoplenib, which has been developed by Korea’s Oscotec. The biopharma has already taken cevidoplenib into a phase 2 study of 60 patients with a rare autoimmune blood disorder called immune thrombocytopenia (ITP).ITP affects around 200,000 people worldwide, including 90,000 in the U.S. alone, according to Agios. The condition is characterized by an immune system that destroys platelets, leading to an increased risk of bleeding.Oscotec’s phase 2 trial missed its primary goal of demonstrating that cevidoplenib could double a patient’s platelet count after 12 weeks of treatment. But Agios was undeterred, pointing to “durable and clinically meaningful platelet responses observed in the cevidoplenib arm compared with placebo across multiple secondary endpoints that align with primary endpoints used in ITP registrational trials.”Agios’ plan is to take the therapy into phase 3 trials in the first half of 2028, once it has completed additional chemistry, manufacturing, and controls (CMC) development work.Many patients with ITP receive steroids or thrombopoietin receptor agonists, such as Amgen’s Nplate or Novartis’ Promacta. Roche’s Rituxan is also used off-label to treat the condition in the U.S., though the antibody comes with some trade-offs when deployed against ITP. Meanwhile, Sanofi’s Wayrilz became the first BTK inhibitor to score an FDA approval for the clotting disease last year.Agios claimed that while current standard-of-care therapies are focused on increasing platelet counts and reducing bleeding risk, they are “often associated with limitations, including delayed onset of response, lack of durable efficacy, and class-specific adverse events, such as decreased blood counts and an increased risk of infections.”In return for the exclusive global rights to cevidoplenib, the Cambridge, Massachusetts-based biopharma is paying $25 million upfront and is liable for up to a further $140 million in development and regulatory milestones across up to three indications in the U.S. and Europe. If the drug secures an approval, Oscotec could also be in line for commercial milestone payments as well as royalties ranging from high single digit to mid-teens.Once the drug has cleared a phase 3 trial, Seongnam-based Oscotec has the option to secure the right to commercialize cevidoplenib on its home turf of South Korea.Cevidoplenib’s focus on ITP falls within Agios’ comfort zone of blood disorders. The biopharma’s sole commercial product is Pyrukynd, a PK activator that won approval in 2022 as the first therapy indicated for hemolytic anemia in adults with PK deficiency and has since expanded to treat anemia in adults with alpha- or beta-thalassemia under the brand name Aqvesme. Last week, Agio’s next-gen PK drug tebapivat failed a phase 2 study in lower-risk myelodysplastic syndromes, but the company is still persevering with the therapy in sickle cell disease.

Phase 2Drug ApprovalPhase 3Immunotherapy

100 Deals associated with Eltrombopag Diolamine

Login to view more data

External Link

KEGG	Wiki	ATC	Drug Bank
D03978	Eltrombopag Diolamine	B02BX05	DB06210

R&D Status

Approved

10 top approved records.

Login

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

Developing

10 top R&D records.

Login

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myelodysplastic Syndromes	Phase 3	Austria	GlaxoSmithKline Research & Development Ltd.	25 Jul 2014
Neoplasms	Phase 3	Austria	GlaxoSmithKline Research & Development Ltd.	25 Jul 2014
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

Login to view more data

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03206086 (CTgov)	Phase 2	Fanconi Anemia	25	Eltrombopag	oakiokqqji = wtoamnfcen mrlymczvuv (pcpofngrvp, ynbjoatcsi - vcqlotethu) View more	-	16 Sep 2026
NCT05438875 (Pubmed \| NEJM Evid)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	96	ATRA plus Eltrombopag	ixuwotcgcz(iszkcifvfk) = obdlsutqem oqrfkgoaha (fpmzgzorzo ) View more	Positive	28 Jul 2026
				Eltrombopag	ixuwotcgcz(iszkcifvfk) = smlmwjhqam oqrfkgoaha (fpmzgzorzo ) View more
ISTH2026	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	3	Eltrombopag and Romiplostim	tzfweuutgi(qghtbioeyg) = npsloacdfc pczxidqdmn (txsepetnsa )	Positive	12 Jul 2026
ISTH2026	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	272	Eltrombopag	pzdccgnech(hogzbavgzr) = including bloating, headache, cough, and weight gain in 10% of patients. fyshafgsrp (xhzrygllmz )	Positive	12 Jul 2026
ISTH2026	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	70	Eltrombopag (Plasma concentration-guided dosing)	snkxjctlnj(gvtrlybiyr): P-Value = 0.04	Positive	12 Jul 2026
				Eltrombopag (Conventional platelet-based dose adjustment)
NCT03413306 (EHA2026)	Phase 3	Refractory Severe Aplastic Anemia	200	Eltrombopag + IST	onvqxsnkoe(myfqjsbvde): P-Value = 0.034 View more	Positive	12 May 2026
				IST
EHA2026	Not Applicable	Purpura, Thrombocytopenic, Idiopathic	120	Eltrombopag	rlptjsdfph(uiuovsfwdh) = lstoezepee qoucsntqfq (onlelxinxs ) View more	Positive	12 May 2026
				Romiplostim	rlptjsdfph(uiuovsfwdh) = euoiuqsxuf qoucsntqfq (onlelxinxs ) View more
EHA2026	Not Applicable	Anemia, Aplastic	40	ATG eltrombopagCSA (pre-eltrombopag era (before 2018))	cehuqasrsi(dmngjkqvsk) = qznvrggzch snpjuvcmap (mrzbbxhrbh )	Positive	12 May 2026
				ATG ±eltrombopagCSA (post-eltrombopag era)	cehuqasrsi(dmngjkqvsk) = lzvourovtz snpjuvcmap (mrzbbxhrbh )
NCT02773225 (EMAA, EHA2026)	Phase 3	Anemia, Aplastic First line	85	Eltrombopag 150 mg/day + Cyclosporine	bordywnfsh(kfzqnoxhjv) = oukvpyzaru xcwigfeaoq (pipovahzdj, 36.14 - NA) View more	Positive	12 May 2026
				Placebo + Cyclosporine	bordywnfsh(kfzqnoxhjv) = bnvkiyanpm xcwigfeaoq (pipovahzdj, 24.00 - 56.14) View more
EHA2026	Not Applicable	Acute Myeloid Leukemia	51	Eltrombopag plus VA	adcsnqrakc(asjgudksot) = dhjublysdv mkwlxfiqwl (cpdqofvwyc, 4.4 - 34.9) View more	Positive	12 May 2026
				VA	adcsnqrakc(asjgudksot) = mnlfcocalo mkwlxfiqwl (cpdqofvwyc, 18.0 - 57.5) View more