Last update 01 Nov 2024

Epoprostenol Sodium

Last update 01 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(5Z,13E)-(15S)-6,9alpha-Epoxy-11alpha,15-dihydroxyprosta-5,13-dienoate, (5Z,9α,11α,13E,15S)-6,9-epoxy-11,15-dihydroxyprosta-5,13-dien-1-oic acid, Caripul

+ [21]

Target

PGI2 receptor

Mechanism

PGI2 receptor agonists(Prostanoid IP receptor agonists), Platelet aggregation inhibitors

Therapeutic Areas

Respiratory DiseasesOther Diseases

Active Indication

Pulmonary Arterial HypertensionHypertension, Pulmonary

Inactive Indication

COVID-19

Originator Organization

GSK Plc

Active Organization

Johnson & Johnson

Actelion Pharmaceuticals Ltd.GlaxoSmithKline LLC

+ [3]

Inactive Organization

Aerogen Pharma Limited

Drug Highest PhaseApproved

First Approval Date

US (20 Sep 1995),

Hypertension, Pulmonary

RegulationOrphan Drug (JP), Priority Review (CN)

Structure

Molecular FormulaC20H32NaO5

InChIKeyWHVPOEPABJZBBY-XQYLJSSYSA-N

CAS Registry61849-14-7

View All Structures (2)

Clinical Trials associated with Epoprostenol Sodium

CTIS2024-512641-16-00 / RecruitingIIT

Efficacy and safety of 72-hour infusion of Prostacyclin (1 ng/kg/min) in mechanically ventilated patients with infectious pulmonary endotheliopathy – a multicentre randomized, placebo-controlled, blinded, investigator-initiated trial” (COMBAT-ARF).

Start Date15 Apr 2024

Sponsor / Collaborator

Rigshospitalet

EUCTR2022-004079-17-DK / Not yet recruitingPhase 2IIT

Efficacy and safety of 72-hour infusion of Prostacyclin (1 ng/kg/min) in mechanically ventilated patients with infectious pulmonary endotheliopathy– a multicentre randomized, placebo-controlled, blinded, investigator-initiated trial - COMBAT-ARF

Start Date20 Mar 2023

Sponsor / Collaborator

Rigshospitalet

NCT04231084 / CompletedPhase 4IIT

Acute Hemodynamic Comparison of Inhaled Nitric Oxide and Inhaled Epoprostenol in Pulmonary Hypertension

The overarching goal of this study is to examine the acute vasoreactive response to both inhaled nitric oxide and inhaled epoprostenol across both traditionally and non-traditionally interrogated phenotypes in PH, and to further characterize the relationship of vasoreactivity to disease severity and PH phenotype.

Start Date15 Jan 2021

Sponsor / Collaborator

The Brigham & Women's Hospital, Inc.

100 Clinical Results associated with Epoprostenol Sodium

100 Translational Medicine associated with Epoprostenol Sodium

100 Patents (Medical) associated with Epoprostenol Sodium

7,935

Literatures (Medical) associated with Epoprostenol Sodium

01 Jan 2025·The Journal of Pediatrics

Patterns and Outcomes of Epoprostenol Use in Infants with Congenital Diaphragmatic Hernia Requiring Extracorporeal Life Support

Article

Author: Shah, Nikhil R. ; Burgi, Keerthi ; McCormick, Amanda D ; McCormick, Amanda D. ; Shah, Nikhil R ; Perrone, Erin E. ; Olive, Mary K. ; Mychaliska, George B. ; Perrone, Erin E ; Lotakis, Dimitra M ; Olive, Mary K ; Lotakis, Dimitra M. ; Church, Joseph T ; Mychaliska, George B ; Church, Joseph T.

OBJECTIVETo describe our experience utilizing epoprostenol for pulmonary hypertension (PH) in infants with congenital diaphragmatic hernia (CDH) requiring extracorporeal life support (ECLS).STUDY DESIGNWe retrospectively reviewed infants diagnosed with CDH who required ECLS at our institution from 2013 to 2023. Data collected included demographics, disease characteristics, medication administration patterns, and hospital outcomes. We first compared infants who received intravenous epoprostenol and those who did not. Among infants who received epoprostenol, we compared survivors and nonsurvivors. χ² test/Fisher's exact and Mann-Whitney tests were used, with significance defined at P < .05.RESULTSFifty-seven infants were included; 40 (70.2%) received epoprostenol. Infants receiving epoprostenol had lower observed/expected total fetal lung volume (O/E TFLV) on magnetic resonance imaging (20 vs 26.2%, P = .042) as well as higher prenatal frequency of liver-up (90 vs 64.7%, P = .023) and "severe" classification (67.5 vs 35.3%, P = .007). Survival with and without epoprostenol was comparable (60% vs 64%, P = .23). Of those receiving epoprostenol, both survivors and nonsurvivors had similar prenatal indicators of disease severity. Most (80%) of hernia defects were classified as type C/D and 68% were repaired <72 hours after ECLS cannulation. The median age at initiation of epoprostenol was day of life 6 (IQR: 4, 7) in survivors and 8 (IQR: 7, 16) in nonsurvivors (P = .012). Survivors had shorter ECLS duration (11 vs 20 days, P = .049). Of nonsurvivors, refractory PH was the cause of death for 13 infants (81%).CONCLUSIONSIn infants with CDH requiring ECLS, addition of epoprostenol appears promising and earlier initiation may affect survival.

01 Dec 2024·Cellular and Molecular Life Sciences

Prostaglandin I2 signaling prevents angiotensin II-induced atrial remodeling and vulnerability to atrial fibrillation in mice

Article

Author: Shi, Xuelian ; Yan, Yingqun ; Li, Guangping ; Sun, Weiyan ; Zhang, Yue ; Yuan, Meng ; Chen, Tienan ; Liu, Tong ; Bao, Qiankun ; Liu, Daiqi ; Zhu, Yi ; Zhang, Bangying ; Cai, Wenbin ; Song, Wenhua ; Zhang, Xu

AbstractAtrial fibrillation (AF) is the most common arrhythmia, and atrial fibrosis is a pathological hallmark of structural remodeling in AF. Prostaglandin I2 (PGI2) can prevent the process of fibrosis in various tissues via cell surface Prostaglandin I2 receptor (IP). However, the role of PGI2 in AF and atrial fibrosis remains unclear. The present study aimed to clarify the role of PGI2 in angiotensin II (Ang II)-induced AF and the underlying molecular mechanism. PGI2 content was decreased in both plasma and atrial tissue from patients with AF and mice treated with Ang II. Treatment with the PGI2 analog, iloprost, reduced Ang II-induced AF and atrial fibrosis. Iloprost prevented Ang II-induced atrial fibroblast collagen synthesis and differentiation. RNA-sequencing analysis revealed that iloprost significantly attenuated transcriptome changes in Ang II-treated atrial fibroblasts, especially mitogen-activated protein kinase (MAPK)-regulated genes. We demonstrated that iloprost elevated cAMP levels and then activated protein kinase A, resulting in a suppression of extracellular signal-regulated kinase1/2 and P38 activation, and ultimately inhibiting MAPK-dependent interleukin-6 transcription. In contrast, cardiac fibroblast-specific IP-knockdown mice had increased Ang II-induced AF inducibility and aggravated atrial fibrosis. Together, our study suggests that PGI2/IP system protects against atrial fibrosis and that PGI2 is a therapeutic target for treating AF.The prospectively registered trial was approved by the Chinese Clinical Trial Registry. The trial registration number is ChiCTR2200056733. Data of registration was 2022/02/12.

01 Dec 2024·The Journal of Pediatrics

Safety and Effectiveness of Selexipag in Pediatric Pulmonary Hypertension: A Retrospective Multicenter Cohort Study

Article

Author: Krishnan, Usha S ; Pan, Zhaoxing ; Frank, Benjamin S ; Ivy, D Dunbar ; Miller-Reed, Kathleen ; Gentzler, Eliza R ; Rosenzweig, Erika B ; Avitabile, Catherine M

OBJECTIVETo describe the safety and effectiveness of treating pediatric patients who have pulmonary arterial hypertension (PAH) with selexipag in a real-world, multi-center cohort, given that data supporting its use in pediatric PAH are sparse.STUDY DESIGNWe report a multi-center, retrospective, cohort study of children with PAH treated with selexipag. Demographic and clinical variables were extracted from the medical records. Clinical parameters were analyzed at 3 timepoints: pre-selexipag, 3-12 months post-selexipag, and >12 months follow-up.RESULTSEighty-seven patients were included, 32 received selexipag as add-on to background therapy, and 55 transitioned from another prostanoid. Median starting and final doses were 4.7 and 28.5 μg/kg/dose BID, respectively. Add-on patients demonstrated improved indexed pulmonary to systemic vascular resistance ratio after selexipag initiation (PVRi/SVRi, 0.62v0.53, p=0.034) with a lower average mean pulmonary artery pressure (MPAP, 46v39 mmHg, p=NS), and oxygen consumption (VO2 max 27.8v30.9 mL/kg/min, p=NS). Transition patients demonstrated stable MPAP (47v45 mmHg, p=NS) and a lower mean PVRi (10.9v8.2 Wood units*m², p=NS) but late functional worsening in some with VO2 max decreased at follow-up (26.0v19.5 ml/kg/min). Side effects were noted in 40% of the cohort but prompted discontinuation in only 2%.CONCLUSIONSIn a large, multi-center cohort, the oral prostacyclin agonist selexipag demonstrates favorable tolerability and effectiveness. Add-on patients demonstrated early hemodynamic improvement. Transition patients demonstrated early stability with risk of late functional worsening, highlighting the importance of ongoing monitoring.

News (Medical) associated with Epoprostenol Sodium

09 Sep 2024

·www.prnewswire.com

Idiopathic Pulmonary Fibrosis Treatment Drug Market to Increase 2 Folds by 2034 | DelveInsight

The emerging therapies for the treatment of idiopathic pulmonary fibrosis include a great deal of emerging drugs Tyvaso (treprostinil), BI 1015550, and others. The emerging therapies in the pipeline have the potential to change the IPF market size and manage the disease burden and progression. LAS VEGAS, Sept. 9, 2024 /PRNewswire/ -- Idiopathic pulmonary fibrosis (IPF) is when the lungs become scarred, and breathing becomes increasingly difficult. Damage to the alveolar epithelium and abnormal wound repair are theorized to be key factors in the development of this disease. Although the cause of IPF is unknown, researchers postulate that the disease probably results from genetic and environmental factors. There is a strong possibility that genetic changes increase a person's risk of developing IPF and subsequent exposure to certain environmental factors that further trigger the disease. However, much is still unknown about this emerging field of study. As per DelveInsight analysis, the total diagnosed prevalent cases of IPF in the 7MM was 193K cases in 2023 which are expected to rise, at a significant CAGR during the study period (2020–2034). The therapeutic approach of IPF involves both nonpharmacological and pharmacological strategies. The goal of the treatment is to slow disease progression, reduce symptoms, prevent acute exacerbations, and prolong survival. Currently, two drugs, OFEV (nintedanib) and ESBRIET (pirfenidone), are approved for treating IPF. These drugs are classified as anti-fibrotic agents, which clinical trials have shown to slow down lung fibrosis or scarring. Pirfenidone is a modified pyridine small molecule with anti-fibrotic, anti-inflammatory, and antioxidant properties. Learn more about the FDA-approved IPF drugs @ Drugs for Idiopathic Pulmonary Fibrosis Treatment Pirfenidone reduces the likelihood of respiratory-related hospitalization over a year of treatment. It is generally well-tolerated, with mild side effects such as skin rash, weight loss, nausea, and fatigue. However, there have been instances of abnormal liver function, including elevated serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), and bilirubin levels, necessitating regular liver function monitoring for patients on pirfenidone. Nintedanib is an intracellular tyrosine kinase inhibitor that binds to adenosine triphosphate binding sites, thus inhibiting the signaling pathways associated with vascular endothelial growth factor receptor, fibroblast growth factor receptor 1–3, and platelet-derived growth factor receptor α and β. The main side effects are diarrhea and nausea, which are manageable with medication, but hepatotoxicity can occur in rare cases. Before May 2022, ESBRIET and OFEV were the only approved treatments for IPF available on the market. However, Sandoz Pharmaceutical recently launched a generic version of pirfenidone in the US. This new entry is expected to significantly impact Hoffmann-La Roche's ESBRIET market and disrupt the market for OFEV as well. Over the years, various drugs have been studied for treating IPF. With the ongoing need for more advanced and specific treatment approaches, new therapies beyond conventional methods are being developed for IPF. To know more about IPF treatment options, visit @ New Treatment for Idiopathic Pulmonary Fibrosis Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years. To date, there are several emerging market players, United Therapeutics, Boehringer Ingelheim, Pliant Therapeutics, Inc., and others that are developing drugs for the treatment of IPF. Discover which therapies are expected to grab major IPF market share @ Idiopathic Pulmonary Fibrosis Market Report Tyvaso (treprostinil), developed by United Therapeutics, is a prostacyclin mimetic approved to treat pulmonary arterial hypertension (PAH; WHO Group 1) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3), aiming to enhance exercise capacity. The company is currently conducting a global Phase III clinical trial called TETON 1 to evaluate Tyvaso for the treatment of patients with IPF. BI 1015550, an experimental oral PDE4B inhibitor with both antifibrotic and anti-inflammatory properties, is under development by Boehringer Ingelheim. Recently, the company released encouraging 12-week Phase II data demonstrating a reduction in lung function decline in IPF patients. The drug is now in Phase III of clinical trials. PLN-74809, the leading program from Pliant Therapeutics, is an oral, small-molecule dual selective inhibitor targeting αVβ1 and αVβ6 integrins, under development for idiopathic pulmonary fibrosis (IPF) and primary sclerosing cholangitis (PSC). These integrins are responsible for the upstream activation of TGF-β1 in fibrotic tissues. By inhibiting these integrins, PLN-74809 aims to block TGF-β1 activation, thus preventing the growth of fibrotic tissue in the lungs and bile ducts. The drug is currently in Phase II of clinical development. Fipaxalparant (HZN-825), developed by Horizon Therapeutics/Amgen, is an oral selective lysophosphatidic acid receptor 1 (LPAR-1) antagonist that has demonstrated promising early clinical results in treating systemic sclerosis. The company is now conducting a Phase II pivotal trial to assess HZN-825 for the treatment of IPF. Discover more about drugs for IPF in development @ Idiopathic Pulmonary Fibrosis Clinical Trials The anticipated launch of these emerging therapies for IPF are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the IPF market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for IPF is expected to grow from USD 3.2 billion in 2023 with a significant CAGR by 2034. This growth can be attributed to the increasing prevalent cases over the globe and thus the surge in treatment options. The expected launch of emerging therapies for IPF treatment is expected to boost the market in the forecast period (2023–2034). DelveInsight's latest published market report titled as Idiopathic Pulmonary Fibrosis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the IPF country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The IPF market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of Idiopathic Pulmonary Fibrosis Gender-specific Diagnosed Prevalent Cases of Idiopathic Pulmonary Fibrosis Age-specific Diagnosed Prevalent Cases of Idiopathic Pulmonary Fibrosis Severity-specific Diagnosed Prevalent Cases of Idiopathic Pulmonary Fibrosis The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM IPF market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this IPF market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the IPF market. Also, stay abreast of the mitigating factors to improve your market position in the IPF therapeutic space. Related Reports Idiopathic Pulmonary Fibrosis Pipeline Idiopathic Pulmonary Fibrosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key idiopathic pulmonary fibrosis companies, including FibroGen, United Therapeutics, Bellerophon Therapeutics, MediciNova, Novartis, Endeavor BioMedicines, Pliant Therapeutics, Nitto Denko, Kadmon Pharmaceuticals, Calliditas Therapeutics, Avalyn Pharmaceuticals, PureTech Health, Taiho Pharmaceutical, Bristol-Myers Squibb, Galecto Biotech AB, CSL Behring, Celgene Pharmaceutical, Vicore Pharma, Boehringer Ingelheim, Guangdong Raynovent, Sunshine Lake Pharma co, Suzhou Zelgen Biopharmaceuticals, Algernon Pharmaceuticals, Horizon Therapeutics, Daewoong Pharmaceutical, Metagone Biotech, AstraZeneca, Lung Therapeutics, Bridge Biotherapeutics, AstraZeneca, Kinarus AG, Insmed, Reviva Pharmaceuticals, Annapurna Bio, Guangdong Hengrui Pharmaceutical Co., Ltd, Ark Biosciences, Ocean Biomedical, among others. Cough in Idiopathic Pulmonary Fibrosis Market Cough in Idiopathic Pulmonary Fibrosis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cough in idiopathic pulmonary fibrosis companies, including NeRRe Therapeutics, Trevi Therapeutics, Algernon Pharmaceuticals, Seyltx Inc., Melius Pharma AB, Cellular Sciences, Emphycorp, among others. Pulmonary Fibrosis Pipeline Pulmonary Fibrosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key pulmonary fibrosis companies, including Roche, Boehringer Ingelheim, Galecto Biotech, Cipla, Gilead Sciences, Fibrogen, Bristol-Myers Squibb, among others. Pulmonary Fibrosis Market Pulmonary Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key pulmonary fibrosis companies, including Roche, Boehringer Ingelheim, Galecto Biotech, Cipla, Gilead Sciences, Fibrogen, Bristol-Myers Squibb, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

Phase 2Drug Approval

04 Sep 2024

·www.biospace.com

WINREVAIR® (sotatercept) is now authorized for use in Canada for Adults with Pulmonary Arterial Hypertension

KIRKLAND, QC , Sept. 4, 2024 /CNW/ - Merck (NYSE: MRK), known as MSD outside of the United States and Canada , announced that WINREVAIR ® (sotatercept) is now authorized for use in Canada in combination with standard pulmonary arterial hypertension (PAH) therapy, for the treatment of adults with World Health Organization [WHO] Group 1 PAH and Functional Class (FC) II or III. Sotatercept is the first activin signaling inhibitor therapy for PAH in Canada . This represents a new class of therapy that works by improving the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation in PAH patients. “We understand the burden that PAH places on patients and caregivers. The approval of WINREVAIR ® represents a step forward for those grappling with this disease. Our commitment to advancing scientific research and developing solutions stands firm to address the unmet needs of Canadians,” said David D. Jones , Managing Director of Merck Canada. About STELLAR The STELLAR study (NCT04576988) was a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which 323 patients with PAH (WHO Group 1 FC II or III) were randomized 1:1 to sotatercept (target dose 0.7 mg/kg) (n=163) or placebo (n=160) plus stable background therapy administered subcutaneously once every 3 weeks. The most common PAH etiologies were idiopathic PAH (59%), heritable PAH (18%), and PAH associated with connective tissue diseases (CTD) (15%). Most participants were receiving either three (61%) or two (35%) background drugs for PAH, and 40% were receiving prostacyclin infusions. The mean time from PAH diagnosis was 8.8 years. Patients had a WHO FC II (49%) or III (51%) at baseline. WINREVAIR ® is the subject of a licensing agreement with Bristol Myers Squibb. About Merck At Merck, known as MSD outside of the United States and Canada , we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable, and healthy future for all people and communities. For more information about our operations in Canada , visit and connect with us on LinkedIn and X @MerckCanada. Forward-Looking Statement of Merck & Co. Inc., Rahway, NJ , USA This news release of Merck & Co., Inc., Rahway, N.J. , USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2023 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site ( ). ® Merck Sharp & Dohme B.V. Used under license. © 2024 Merck & Co., Inc., Rahway, NJ , USA and its affiliates. All rights reserved. SOURCE Merck Canada Inc.

License out/inDrug ApprovalClinical Result

26 Aug 2024

·pipelinereview.com

Merck Receives European Commission Approval for WINREVAIR™ (sotatercept) in Combination With Other Pulmonary Arterial Hypertension (PAH) Therapies, for the Treatment of PAH in Adult Patients With Functional Class II-III

WINREVAIR is the first activin signaling inhibitor therapy for PAH approved in Europe RAHWAY, NJ, USA I August 26, 2024 I Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the European Commission (EC) has approved WINREVAIR™ (sotatercept), in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH in adult patients with World Health Organization (WHO) Functional Class (FC) II to III, to improve exercise capacity. WINREVAIR is the first and only activin signaling inhibitor therapy for PAH approved in all 27 member states of the EU, as well as Iceland, Liechtenstein and Norway. WINREVAIR works by improving the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation underlying PAH. The EC approval of WINREVAIR is based on safety and efficacy results from the Phase 3 STELLAR trial. “The European Commission’s approval of WINREVAIR is an important step for patients,” said Dr. Joerg Koglin, senior vice president and head of general medicine, global clinical development, Merck Research Laboratories. “WINREVAIR is the first therapy targeting the activin signaling pathway. We are proud to bring this innovative treatment to more patients and remain committed to further investigating the potential of WINREVAIR in areas where there are unmet needs in the management of PAH.” “Pulmonary arterial hypertension is a devastating disease for patients, who suffer from debilitating symptoms that can severely limit their daily activities,” said Dr. Marc Humbert, Professor of Medicine and Director of the Pulmonary Hypertension Reference Center at Université Paris-Saclay. “New treatment opinions continue to be needed for patients. Based on the Phase 3 STELLAR study, adding WINREVAIR to background PAH therapy improved exercise capacity, reduced the risk of death or clinical worsening events and improved functional class compared to background PAH therapy alone. These findings are significant and reinforce that WINREVAIR, in combination with other PAH therapies, should be considered as a new standard of care for the treatment of functional class II and III adult patients.” The EC approval is based on the Phase 3 STELLAR trial, which compared WINREVAIR (n=163) to placebo (n=160), both in combination with background standard of care therapies in adult patients with PAH (WHO Group 1, FC II or III). The primary efficacy endpoint was change from baseline at Week 24 in six-minute walk distance. Treatment with WINREVAIR resulted in a statistically significant and clinically meaningful improvement in six-minute walk distance of 40.8 meters over placebo (95% CI: 27.5, 54.1; p<0.001). WINREVAIR also significantly improved multiple important secondary outcome measures, including reducing the risk of death or clinical worsening. In a post hoc analysis provided to EMA, time to death or clinical worsening was defined as the time from randomization to the first occurrence of deterioration of PAH, PAH-specific hospitalization, worsening-related listing for lung and/or heart transplant, need for atrial septostomy, or death from any cause. There was an 82% reduction in the risk of death or clinical worsening with WINREVAIR on top of background therapy versus background therapy alone (number of events: 7 vs 29, HR=0.182; 95% CI: 0.075, 0.441; p<0.001). WINREVAIR is administered once every 3 weeks as a single injection under the skin and may be administered by patients or caregivers with guidance, training and follow-up from a healthcare provider. Healthcare providers and patients/caregivers should refer to the Instructions for Use for information on the proper preparation and administration of WINREVAIR. This approval by the EC for WINREVAIR is valid in all 27 member states of the EU, as well as Iceland, Liechtenstein and Norway. WINREVAIR was previously granted Priority Medicines (PRIME) and orphan designation by the EMA for the treatment of PAH. On March 26, 2024, the FDA approved WINREVAIR in the U.S. for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events. About STELLAR The STELLAR study (NCT04576988) was a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial in which 323 patients with PAH (WHO Group 1 FC II or III) were randomized 1:1 to WINREVAIR (target dose 0.7 mg/kg) (n=163) or placebo (n=160) plus stable background therapy administered subcutaneously once every 3 weeks. The most common PAH etiologies were idiopathic PAH (59%), heritable PAH (18%), and PAH associated with connective tissue diseases (CTD) (15%). Most participants were receiving either three (61%) or two (35%) background drugs for PAH, and 40% were receiving prostacyclin infusions. The mean time from PAH diagnosis was 8.8 years. Patients had a WHO FC II (49%) or III (51%) at baseline. About WINREVAIR™ (sotatercept-csrk) for injection, for subcutaneous use, 45 mg, 60 mg WINREVAIR, the first activin signaling inhibitor therapy approved to treat PAH, improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, WINREVAIR induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodeling, and improved hemodynamics. WINREVAIR is the subject of a licensing agreement with Bristol Myers Squibb. Selected Safety Information for WINREVAIR in the U.S. WINREVAIR may increase hemoglobin and lead to erythrocytosis. Severe erythrocytosis may increase the risk of thromboembolic events or hyperviscosity syndrome. Monitor Hgb before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter, to determine if dose adjustments are required. WINREVAIR may decrease platelet count and lead to severe thrombocytopenia, which may increase the risk of bleeding; thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Do not initiate treatment if platelet count is <50,000/mm 3 . Monitor platelets before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required. In clinical studies, serious bleeding (e.g., gastrointestinal, intracranial hemorrhage) was reported in 4% of patients taking WINREVAIR and 1% of patients taking placebo. Serious bleeding was more likely in patients on prostacyclin background therapy and/or antithrombotic agents, or with low platelet counts. Advise patients about signs and symptoms of blood loss. Do not administer WINREVAIR if the patient is experiencing serious bleeding. WINREVAIR may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment with WINREVAIR and for at least 4 months after the final dose. Pregnancy testing is recommended for females of reproductive potential before starting WINREVAIR treatment. Based on findings in animals, WINREVAIR may impair female and male fertility. Advise patients on the potential effects on fertility. The most common adverse reactions occurring in the Phase 3 clinical trial (≥10% for WINREVAIR and at least 5% more than placebo) were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%), rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea (15.3% vs 10.0%), dizziness (14.7% vs 6.2%), and erythema (13.5% vs 3.1%). Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with WINREVAIR, and for 4 months after the final dose. About pulmonary arterial hypertension (PAH) Pulmonary arterial hypertension (PAH) is a rare, progressive and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. Approximately 40,000 people in the U.S. and 30,000 people in the EU are living with PAH. The disease progresses rapidly for many patients. PAH results in significant strain on the heart, leading to limited physical activity, heart failure and reduced life expectancy. The five-year mortality rate for patients with PAH is approximately 43%, based on data from the REVEAL registry (patients enrolled between March 2006 and December 2009). About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter) , Facebook , Instagram , YouTube and LinkedIn . SOURCE: Merck

Clinical ResultPhase 3Drug ApprovalOrphan Drug

100 Deals associated with Epoprostenol Sodium

External Link

KEGG	Wiki	ATC	Drug Bank
D01337	Epoprostenol Sodium	B01AC09	DB01240

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hypertension, Pulmonary	US	GlaxoSmithKline LLC	20 Sep 1995
Pulmonary Arterial Hypertension	JP	Actelion Pharmaceuticals Ltd.	-

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
COVID-19	Phase 2	US	Aerogen Pharma Limited	15 Sep 2020
Hypertension, Pulmonary	Phase 2	US	Aerogen Pharma Limited	23 Aug 2017
Pulmonary Arterial Hypertension	Phase 2	BE	Actelion Pharmaceuticals US, Inc.	01 Mar 2011
Pulmonary Arterial Hypertension	Phase 2	CA	Actelion Pharmaceuticals US, Inc.	01 Mar 2011
Pulmonary Arterial Hypertension	Phase 2	IT	Actelion Pharmaceuticals US, Inc.	01 Mar 2011
Pulmonary Arterial Hypertension	Phase 2	ES	Actelion Pharmaceuticals US, Inc.	01 Mar 2011
Pulmonary Arterial Hypertension	Phase 2	FR	Actelion Pharmaceuticals US, Inc.	01 Mar 2011
Pulmonary Arterial Hypertension	Phase 2	NL	Actelion Pharmaceuticals US, Inc.	01 Mar 2011

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02251041 (CAPITL, CTgov)	Phase 2	Reperfusion Injury	143	C1-Inhibitor+Epoprostenol+Infliximab+Melatonin+Alfa-tocopherol+Human recombinant erythropoietin+Glutathione	jdwwetufxc(zqobufwzdx) = qockguastb qftiwpvwkg (twgxbcttuv, xdwrjelcxy - ljecsrgmwn) View more	-	23 Aug 2024
NCT03081052 (Pubmed)	Phase 4	-	231	Inhaled Epoprostenol	rzaqcykyhw(rhavlldglo) = kfocnkvodz abakzacipg (doojwunffv )	-	04 Jul 2023
				Nitric Oxide	rzaqcykyhw(rhavlldglo) = iwopnsqwct abakzacipg (doojwunffv )
NCT03081052 (INSPIRE-FLO, CTgov)	Phase 4	Lung Diseases \| Heart Failure \| Graft Rejection	519	iNO (Lung Transplant With iNO)	mdjyblebpc(huxstukchz) = nxuelgwogl meqjhccixz (qzpkeskvsj, ngstmeszzc - ksoewmibgv) View more	-	10 Mar 2022
				iEPO (Lung Transplant With iEPO)	mdjyblebpc(huxstukchz) = vzypmcergh meqjhccixz (qzpkeskvsj, nlhnvjamqt - bhmwiznfsp) View more
NCT01717209 (CTgov)	Phase 4	Heart Failure	14	Nitric Oxide+Prostacyclin	pirtkabdrv(fklzjbybrq) = beqdvbbuuh rcpeboeaux (ugpxorlrfn, yvaikahcqh - nhvmmpztmp) View more	-	09 Oct 2017
NCT02705807 (CTgov)	Phase 4	Cardiovascular Diseases \| Pulmonary Arterial Hypertension	10	Par+epoprostenol sodium	gamxyuxxwt(anxuzacaaf) = zdkpermxvg oicxpolatk (yaaepxnaly, zgmbazrbsj - izrmuqfvum) View more	-	06 Apr 2017
NCT01470144 (Epitome2ext, CTgov)	Phase 3	Pulmonary Arterial Hypertension	41	Epoprostenol	(iqpspqeodc) = xmikfvkdxo xtpfhxpjys (okphqyjhih, igzkugliyn - enrrhgaeta) View more	-	09 Dec 2016
EHA2015	Not Applicable	Myelodysplastic Syndromes	107	ERYTHROPOIETIN (No support)	(gvzhdplwwd) = sydpqzhxsx jvoasqspbd (qohomcxckm ) View more	-	21 May 2015
				Epo support	(gvzhdplwwd) = udlpccyhlm jvoasqspbd (qohomcxckm ) View more
NCT01431716 (EPITOME-2, CTgov)	Phase 3	Pulmonary Arterial Hypertension	42	EFI	zethrbfpbu(wgnzzmghfe) = gcvjxtlsto kfifnjnntx (gtbfkbiuij, fbpneqhkda - itrsitpooa) View more	-	13 Jan 2015
ACR2013	Not Applicable	Interstitial lung disease due to systemic disease	99	Prostacyclin therapy within 6 months of RHC	rysfbtsxbl(lqzunpwvbf) = swxwirygbj xgfdiwhhew (ukagatzoro )	Positive	25 Oct 2013
				Prostacyclin therapy after 6 months of RHC	rysfbtsxbl(lqzunpwvbf) = bsdzzmvfqk xgfdiwhhew (ukagatzoro )
NCT01105117 (EPITOME-1 Ext, CTgov)	Phase 4	Pulmonary Arterial Hypertension	2	ACT-385781A (ACT-385781A (Epoprostenol for Injection))	vkgpwycwrp(lquycnjfox) = wgncfbfixw yvhodipols (tlesnqrgfd, mauminqsot - yhkjzstihr) View more	-	23 Aug 2012
				Flolan (Flolan® (Epoprostenol Sodium) for Injection)	vkgpwycwrp(lquycnjfox) = qqroczknjl yvhodipols (tlesnqrgfd, pwrowdywxg - okvygkwrqp) View more

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