Delving into the Latest Updates on Iloprost with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Breelib, Ciloprost, Endoprost

+ [27]

Target

PGI2 receptor

Action

agonists

Mechanism

PGI2 receptor agonists(Prostanoid IP receptor agonists)

Therapeutic Areas

Cardiovascular DiseasesRespiratory DiseasesOther Diseases+ [2]

Active Indication

ChilblainsFrostbitePulmonary Arterial Hypertension+ [5]

Inactive Indication

Acute Lung InjuryAsthmaCREST Syndrome+ [15]

Originator Organization

Actelion Pharmaceuticals Ltd.

Active Organization

BTG International, Inc.Bayer Australia Ltd.Actelion Pharmaceuticals US, Inc.

+ [4]

Inactive Organization

Thrombologic ApS

CiVi Biopharma, Inc.

Actelion Pharmaceuticals Ltd.

+ [3]

License Organization

CiVi Biopharma, Inc.

Bayer AG

Advanz Pharma Corp. Ltd.

Drug Highest PhaseApproved

First Approval Date

European Union (15 Sep 2003),

Familial Primary Pulmonary Hypertension

RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (South Korea), Priority Review (United States)

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Structure/Sequence

Molecular FormulaC22H32O4

InChIKeyHIFJCPQKFCZDDL-ACWOEMLNSA-N

CAS Registry78919-13-8

Study Title: A Clinical Study of Inhaled Iloprost for the Treatment of Pulmonary Hypertension in Patients With Chronic Obstructive Pulmonary Disease (HOLLYWOOD)
The goal of this clinical study is to learn if the inhaled drug iloprost is effective and safe for treating pulmonary hypertension (PH) in adult patients who have severe or very severe Chronic Obstructive Pulmonary Disease (COPD).
The main questions it aims to answer are:
Does inhaled iloprost reduce the pressure and resistance in the lung's blood vessels (measured as Pulmonary Vascular Resistance - PVR)?
Does inhaled iloprost improve participants' ability to exercise, measured by how far they can walk in 6 minutes?
What are the side effects and medical problems that participants experience while taking inhaled iloprost?
Researchers will assess changes in participants' health by comparing measurements taken before they start taking inhaled iloprost to measurements taken after 12 weeks of treatment. There is no placebo group in this study.
Participants in this study will:
Use an inhaler to take iloprost 6 to 9 times every day for 12 weeks.
Visit the clinic for checkups at the beginning of the study and after the 12-week treatment period.
Undergo tests including an exercise capacity test (the 6-minute walk test) and heart pressure measurements (hemodynamic tests) before and after the treatment period.

Start Date10 Dec 2025

Sponsor / Collaborator

University of Sao Paulo General Hospital

NCT05411107

/ WithdrawnPhase 2IIT

Phase II Trial of Oral Iloprost for the Precision Chemoprevention of Lung Cancer

This phase II trial tests whether oral iloprost works in preventing lung cancer (chemoprevention) in former smokers. Oral iloprost has previously been shown to reduce abnormal lung cells in former smokers, suggesting a clinically significant impact on lung cancer risk. The use of oral iloprost may help keep cancer from forming and reduce abnormal cells in the lung in order to lower the risk of developing lung cancer in former smokers.

Start Date01 Jun 2024

Sponsor / Collaborator

National Cancer Institute

NCT06319274

/ RecruitingPhase 2IIT

Efficacy and Safety of 72-hour Infusion of Prostacyclin (1 ng/kg/Min) in Mechanically Ventilated Patients With Infectious Pulmonary Endotheliopathy - a Multicenter Randomized, Placebo-controlled, Blinded, Investigator-initiated Trial

The purpose of this clinical trial is to investigate the efficacy and safety of continuous intravenous administration of low dose iloprost versus placebo for 72-hours, in 450 mechanically ventilated patients with infectious respiratory failure. The study hypothesis is that iloprost may be beneficial as an endothelial rescue treatment as it is anticipated to deactivate the endothelium and restore vascular integrity in patients suffering from respiratory failure caused by endothelial breakdown, ultimately improving survival.

Start Date15 Apr 2024

Sponsor / Collaborator

Rigshospitalet

100 Clinical Results associated with Iloprost

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100 Translational Medicine associated with Iloprost

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100 Patents (Medical) associated with Iloprost

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2,663

Literatures (Medical) associated with Iloprost

01 Mar 2026·Toxicon: X

Vascular insufficiency in the extremities following jellyfish-sting envenomation in Malaysia

Article

Author: Tan, Lay Tin ; Ismail, Ahmad Khaldun ; Kamsani, Nur Afiqah ; Khalid, Mohamad Muhyiddin

Introduction:

Vascular insufficiency is an uncommon but potentially limb-threatening complication of jellyfish envenomation, with limited documentation in Southeast Asia. This study characterizes its epidemiology, clinical course, and response to targeted vasodilator therapy in Malaysia.

Methods:

A retrospective cohort review was conducted for confirmed jellyfish-sting cases complicated by vascular insufficiency reported to the Remote Envenomation Consultancy Services (RECS) from 2017 to 2022. Demographic, geographic, clinical, and management data were extracted from RECS consultation logs. Serial Doppler ultrasound findings and treatment responses were analysed descriptively.

Results:

Among 105 jellyfish sting consultations, four (3.8 %) cases of vascular insufficiency were identified, all occurring in coastal Penang and affecting children aged 7-12 years. All primary stings involved the upper limbs; two had concurrent lower limb lesions. The onset of peripheral numbness and cyanosis occurred on day three post-sting. Doppler ultrasonography revealed subcutaneous oedema, reduced arterial calibre, and diminished flow velocities consistent with arterial vasospasm. All patients received intravenous iloprost (0.5-2 ng/kg/min) with gradual tapering, guided by clinical and Doppler parameters. Rapid improvement in perfusion was documented in all cases, with minimal adverse effects (vomiting, dyspnoea, haematuria). Hospitalization lasted 12-32 days. Three patients achieved full functional recovery; one had residual scarring and contracture. No deaths occurred.

Conclusion:

Delayed-onset arterial vasospasm can complicate paediatric jellyfish stings, particularly in the upper limb. Early recognition of evolving ischemic signs and timely initiation of iloprost with structured tapering may avert tissue loss. Broader drug availability and standardized treatment algorithms are needed to optimize outcomes in resource-limited coastal settings.

01 Dec 2025·Clinical Respiratory Journal

Endothelial‐Derived Extracellular Vesicles During Exercise in COPD Patients

Article

Author: Johnson, Jessica L. ; DeBoisblanc, Bennett ; Wyczechowska, Dorota ; Lammi, Matthew R. ; Ghonim, Mohamed A. ; Okpechi, Samuel C. ; Bauer, Natalie ; Boulares, Hamid A.

ABSTRACT:

Introduction:

There are no treatments directly targeting the pulmonary vasculature in chronic obstructive pulmonary disease (COPD), and further characterization of the underlying endothelial cell (EC) abnormalities could be helpful in drug development.

Methods:

We investigated the influence of exercise and the prostacyclin analog iloprost on extracellular vesicles derived from ECs (eEVs) in 15 moderate–severe COPD patients who were enrolled in a randomized, placebo‐controlled crossover trial of iloprost.

Results:

Active smokers had a profile consistent with inflammatory‐derived EVs, while exacerbation‐prone COPD subjects had a profile consistent with apoptosis‐derived eEVs. There were no significant effects of iloprost on eEV levels. However, there was a significant increase in CD144+ and CD31+/CD144+ EVs 1 h after exercise.

Conclusions:

Endothelial‐derived EV profiles differed based on smoking and exacerbation history. Iloprost did not affect eEV levels, although maximal exercise induced a delayed increase in a subset of eEVs, possibly through shear stress.

01 Nov 2025·CARDIOLOGY IN THE YOUNG

Inhaled iloprost and 2D phase-contrast cardiac MRI in pulmonary vascular reversibility: an adolescent case of anomalous right pulmonary artery from the ascending aorta

Article

Author: Bilal, Mehmet Salih ; Çeliker, Alpay ; Kızılkaya, Mete Han ; Gumus, Terman

Abstract:

The anomalous origin of the right pulmonary artery from the ascending aorta is a rare congenital anomaly usually diagnosed in infancy. When diagnosed later during adolescence, it presents diagnostic and treatment challenges, especially in determining whether pulmonary vascular disease can be reversed. We report the case of an 11-year-old girl who experienced increasing fatigue and was diagnosed with anomalous origin of the right pulmonary artery from the ascending aorta through echocardiography, CT angiography, and cardiac catheterisation. Segmental pulmonary hypertension was noted, but the operability was uncertain. A new non-invasive test combining 2D phase-contrast cardiac MRI with inhaled iloprost was performed. Flow measurements revealed a 42% rise in right pulmonary artery flow, indicating preserved vasoreactivity. Lung biopsy confirmed pulmonary vascular changes consistent with Heath–Edwards Stage II–III. Based on these findings, surgical reimplantation of the anomalous artery into the main pulmonary artery was performed. The postoperative recovery after surgery was uneventful, and follow-up catheterisation demonstrated normalised haemodynamics without residual stenosis. This case highlights the potential value of cardiac MRI–based vasoreactivity testing with inhaled iloprost as an additional tool alongside standard diagnostics for evaluating operability in late-presenting anomalous origin of the right pulmonary artery from the ascending aorta. Combining imaging, histopathology, and haemodynamic data allowed a personalised and safe surgical approach.

11

News (Medical) associated with Iloprost

17 Feb 2026

·www.biospace.com

Essential Pharma strengthens rare disease portfolio with acquisition of Ventavis® (iloprost trometamol) for adult primary pulmonary hypertension

Ventavis® is an established inhaled prostacyclin analogue for the treatment of primary pulmonary hypertension Acquisition reinforces Essential Pharma’s mission to serve the needs of rare disease populations and enhances reputation as a trusted partner for continued access to niche medicines Egham, UK – 17 February 2026 – Essential Pharma (“Essential” or “the Company”), a global specialty pharmaceutical company, today announces the acquisition of Ventavis® (iloprost trometamol) from Bayer AG, including rights to the accompanying Breelib® nebuliser delivery technology. The acquisition represents a significant milestone as Essential Pharma continues to grow its specialty rare disease franchise, reinforcing the Company’s mission to deliver medicines for small, underserved or rare disease populations globally. Ventavis®, a prostacyclin analogue, is a well-established treatment for adult patients with primary pulmonary hypertension classified as NYHA functional class III, a rare condition where blood pressure is too high in the blood vessels between the heart and the lungs. Ventavis® is used to improve exercise capacity and symptoms. It is administered through a nebuliser, such as Breelib®, for targeted delivery of the drug to the lungs where it can work most effectively, resulting in improved blood flow, enhanced supply of oxygen to the body and reduced strain on the heart. The transaction expands Essential’s commercial presence in rare cardio-pulmonary disease, together with the acquisition of Colobreathe® announced in early 2024, and develops the Company’s position in rare disease more generally. Under the terms of the acquisition, Essential Pharma has acquired rights to Ventavis® across all global markets, excluding the USA and Russia, and global rights to Breelib®. The product is commercially available in over 60 markets globally. Simon Ball, President Rare Disease and Interim CEO of Essential Pharma, commented : “Adding Ventavis® and the Breelib® nebuliser to Essential Pharma’s growing rare disease portfolio signals our commitment to enabling continued access to treatments that make a difference to individuals living with niche and rare medical conditions. This acquisition leverages our global commercial footprint and highlights our motivation to invest further into rare diseases, building on our strong reputation as a partner of choice in the biopharmaceutical sector.” About Essential Pharma Essential Pharma is a global pharmaceutical company with a mission to develop and deliver medicines for small, underserved or rare disease populations globally. We have a proven track record of acquiring, investing in and commercialising specialty medicines. Our growing product portfolio reaches patients in approximately 70 countries across a number of therapy areas with a particular focus on rare disease, CNS, ophthalmology and gastroenterology. Our investments into rare diseases include the fields of rare cardio-pulmonary and rare paediatric oncology, where our first development-stage asset is an anti-GD2 antibody for the treatment of high-risk neuroblastoma. For more information, visit CONTACTS Essential Pharma Simon Ball, President Rare Disease and Interim CEO Tel: +44 (0) 1784 477 167 Email: info@essentialpharmaceuticals.com ICR Healthcare Tracy Cheung/Chris Welsh Tel: +44 (0) 20 3709 5700 Email: Essentialpharma@icrhealthcare.com

AcquisitionDrug Approval

16 Feb 2024

·firstwordpharma.com

Eicos’ Aurlumyn bags FDA approval as first-ever severe frostbite treatment

The FDA approved Eicos Sciences’ injectable vasodilator Aurlumyn (iloprost) for the treatment of severe frostbite in adults, making it the first drug to be authorised in this indication.“Having this new option provides physicians with a tool that will help prevent the life changing amputation of one’s frostbitten fingers or toes,” remarked Norman Stockbridge, director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research. The approval was supported by findings from a study involving 47 adults with severe frostbite, all having received intravenous aspirin and standard care. Participants were randomly assigned to receive Aurlumyn alone, or other medications that are unapproved for frostbite either alone or in combination with Eicos’ drug. The primary endpoint was a bone scan performed seven days after the onset of frostbite, with the goal of establishing the need for amputation of at least one finger or toe.At day seven, none of the patients given Aurlumyn alone had a bone scan indication for amputation, compared with 19% in those administered Eicos’ drug in combination with other medicines and 60% in the third group. In addition, the Aurlumyn groups had significantly fewer patients with bone scan abnormalities, and the majority of patients’ subsequent amputation status at follow-up was consistent with the bone scan findings. The FDA noted that Aurlumyn’s label carries a warning and precaution highlighting a risk of symptomatic hypotension. Iloprost gained its first FDA approval back in 2004 as an inhaled therapy for the treatment of pulmonary arterial hypertension under the brand Ventavis. Eicos, a subsidiary of CiVi Biopharma, has reformulated the drug into an intravenous form and is also investigating it for systemic sclerosis and as an anticoagulant in patients receiving extracorporeal membrane oxygenation.

Drug ApprovalClinical Result

15 Feb 2024

·www.pharmaceutical-technology.com

FDA approves Eicos’s Aurlumyn for severe frostbite treatment

Aurlumyn injection is a new treatment option for adults with severe frostbite. Credit: movchanzemtsova / Shutterstock.com. The US Food and Drug Administration (FDA) has approved Eicos Sciences’s Aurlumyn (iloprost) injection, a new treatment for adults with severe frostbite to lower the risk of amputation of fingers or toes. Aurlumyn contains iloprost, a vasodilator that opens blood vessels and stops blood clotting, as its active ingredient. The efficacy of iloprost in treating severe frostbite was established through an open-label controlled trial in 47 adult subjects. Participants were randomised into three groups, with group one receiving iloprost intravenously for six hours daily for up to eight days along with aspirin and standard of care. Groups two and three were administered other medications, with and without iloprost, which are not approved for frostbite treatment. See Also: Otsuka wins FDA breakthrough therapy designation for IgAN drug QBiotics wins FDA orphan status for rainforest-derived cancer drug The trial’s primary efficacy measure was bone scans on day seven following the frostbite incident to predict the need for amputation. The scans showed that none of the subjects who received iloprost alone (group one) required amputation compared to 19% in group two and 60% in group three. This indicated a significant reduction in the presence of bone scan abnormalities in groups receiving iloprost. Most subjects had follow-up data available to confirm whether they underwent amputation. This aligned with the bone scan findings. Headache, flushing, heart palpitations, nausea and hypotension were common side effects linked to Aurlumyn treatment. The treatment carries a warning for potential symptomatic hypotension. Aurlumyn was granted priority review and orphan drug designations for this indication, highlighting its significance in addressing severe frostbite, a condition that can escalate from mild to severe, potentially leading to permanent tissue damage and the cessation of blood flow. Iloprost originally received approval in 2004 for pulmonary arterial hypertension. FDA Center for Drug Evaluation and Research Division of Cardiology and Nephrology director Norman Stockbridge stated: “This approval provides patients with the first-ever treatment option for severe frostbite. “Having this new option provides physicians with a tool that will help prevent the life-changing amputation of one’s frostbitten fingers or toes.”

Drug ApprovalOrphan DrugBreakthrough TherapyClinical ResultPriority Review

100 Deals associated with Iloprost

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External Link

KEGG	Wiki	ATC	Drug Bank
D02721	Iloprost	B01AC11	DB01088

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Chilblains	United States	Actelion Pharmaceuticals US, Inc.	14 Feb 2024
Frostbite	United States	BTG International, Inc.	13 Feb 2024
Pulmonary Arterial Hypertension	United States	Actelion Pharmaceuticals US, Inc.	29 Dec 2004
Associated Pulmonary Arterial Hypertension	Australia	Bayer Australia Ltd.	21 Jan 2004
Idiopathic pulmonary arterial hypertension	Australia	Bayer Australia Ltd.	21 Jan 2004
Pulmonary Embolism	Australia	Bayer Australia Ltd.	21 Jan 2004
Familial Primary Pulmonary Hypertension	European Union	Bayer AG	15 Sep 2003
Familial Primary Pulmonary Hypertension	Iceland	Bayer AG	15 Sep 2003
Familial Primary Pulmonary Hypertension	Liechtenstein	Bayer AG	15 Sep 2003
Familial Primary Pulmonary Hypertension	Norway	Bayer AG	15 Sep 2003

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
CREST Syndrome	Phase 3	United States	CiVi Biopharma, Inc.	14 Oct 2019
Raynaud Disease	Phase 3	United States	CiVi Biopharma, Inc.	14 Oct 2019
Scleroderma, Systemic	Phase 3	United States	CiVi Biopharma, Inc.	14 Oct 2019
Heart Failure	Phase 3	United Kingdom	Actelion Pharmaceuticals US, Inc.	20 Apr 2006
Pneumonia, Bacterial	Phase 2	Denmark	Thrombologic ApS	01 Jun 2012
Pneumonia, Bacterial	Phase 2	Finland	Thrombologic ApS	01 Jun 2012
Sepsis	Phase 2	Denmark	Thrombologic ApS	01 Jun 2012
Sepsis	Phase 2	Finland	Thrombologic ApS	01 Jun 2012
Pulmonary Disease, Chronic Obstructive	Phase 2	United States	Actelion Pharmaceuticals US, Inc.	01 Mar 2012
Pulmonary Disease, Chronic Obstructive	Phase 2	France	Actelion Pharmaceuticals US, Inc.	01 Mar 2012

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03788837 (EUCTR2018-001709-10-IT, Pubmed \| Am J Respir Crit Care Med)	Phase 3	Shock, Septic	236	Iloprost	oztyamtyqr(wkfqekwekj) = ghifbyxvvm ozyiterach (hlxbhcsccm )	Negative	01 Jul 2025
				Placebo	oztyamtyqr(wkfqekwekj) = sjbevucvlu ozyiterach (hlxbhcsccm )
NCT03867097 (CTgov)	Phase 2	CREST Syndrome \| Raynaud Disease \| Scleroderma, Systemic	34	Placebo IV infusion (Placebo)	jnqxsohhib(iloyutsbbw) = jvnkxizcjm mwljqorbdj (mupftwrdgo, ykwqfryrjm - wwsvcyydmx) View more	-	25 May 2025
				Iloprost (Iloprost Injection, for Intravenous Use)	jnqxsohhib(iloyutsbbw) = arztzxtqph mwljqorbdj (mupftwrdgo, sszpkcibmd - hekmaxkqbs) View more
NCT04040322 (CTgov)	Phase 3	CREST Syndrome \| Raynaud Disease \| Scleroderma, Systemic	198	Placebo IV infusion (Placebo)	ruvhrfbtec(lbnaraitxl) = clfzccmdcv numqhwjzdz (jopeymsior, loplvopovs - bwfjcchqmn) View more	-	25 May 2025
				Iloprost (Iloprost Injection, for Intravenous Use)	ruvhrfbtec(lbnaraitxl) = uuhrfdxups numqhwjzdz (jopeymsior, grfzzwdigy - cjtzzshzyc) View more
NCT03044314 (OVATION, CTgov)	Phase 4	Hypertension, Pulmonary	27	nitric oxide+Iloprost	oynfewfgkz(hagsnalazs) = iblplafyrb lbzpczdtij (mqnwfzmidc, wncowsuozq - qrofpvhbth) View more	-	02 Apr 2024
NCT04598191 (Pubmed \| Can Anaesth Soc J)	Not Applicable	-	100	Inhaled iloprost (20 μg)	uuewvhridh(gauwjqdcrg) = vzxzmccnkg jiuxxxyrpw (jsnlbftoow, 3)	-	01 Apr 2024
				Saline	uuewvhridh(gauwjqdcrg) = ivkrlhcoui jiuxxxyrpw (jsnlbftoow, 3)
FDA Manual	Not Applicable	Frostbite	47	buflomedil+aspirin+buflomedil (Groups A)	ikbtsnbrsd(brnbrzdowu) = ptlgcrszdf cckbxwonku (juybxvbgio )	Positive	13 Feb 2024
				iloprost+aspirin+buflomedil (Groups B)	ikbtsnbrsd(brnbrzdowu) = qfxecizthv cckbxwonku (juybxvbgio )
NCT02237183 (Phase Ib trial of inhaled iloprost for the prevention of lung cancer with predictive and response biomarker assessment, Pubmed \| Front Oncol)	Phase 1	Lung Cancer interferon γ signaling	-	Inhaled iloprost (5ug) QID	jllgobbsgh(vaoumoqgwa) = svntrgraig ctonvwtwzx (fwkvmxwqpx )	Positive	30 Aug 2023
ATS2023	Not Applicable	Hypertension, Pulmonary	-	Inhaled Iloprost	zdvblecwfn(vpidsqhruc) = drevprcmvk qwwmdlkbew (ctzbvlataj, 12.2)	-	21 May 2023
				Inhaled Nitric Oxide	zdvblecwfn(vpidsqhruc) = vdmlnyxdjg qwwmdlkbew (ctzbvlataj, 12.2)
NCT03293407 (VENTASTEP, Pubmed \| J Med Internet Res)	Not Applicable	Pulmonary Arterial Hypertension	31	Inhaled Iloprost	eqyprmeazp(rvbnxfbesn) = muyawvwwjt jkjgikypru (jnukviinmy ) View more	-	08 Oct 2021
NCT00927654 (ILOCARD, Pubmed \| Can Anaesth Soc J)	Phase 3	-	253	Iloprost	ofbsqkaovx(wyyqjjzkzx) = cimxiwiyjy tlakbfhfjz (wxnuqxassm )	Negative	01 Aug 2019
				Placebo	ofbsqkaovx(wyyqjjzkzx) = ofmbqhgjmb tlakbfhfjz (wxnuqxassm )