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+ [14]

Originator Organization

Actelion Pharmaceuticals Ltd.

Active Organization

Bayer Australia Ltd.Actelion Pharmaceuticals US, Inc.

Pharmosa Biopharm, Inc.

+ [4]

Inactive Organization

License Organization

Essential Pharmaceuticals Ltd.

Bayer AG

+ [1]

Drug Highest PhaseApproved

First Approval Date

European Union (15 Sep 2003),

Familial Primary Pulmonary Hypertension

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

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Structure/Sequence

Molecular FormulaC22H32O4

InChIKeyHIFJCPQKFCZDDL-ACWOEMLNSA-N

CAS Registry78919-13-8

Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS). It primarily affects individuals from Africa or the French overseas territories. In France, approximately 26,000 patients are affected. Improved care has significantly increased life expectancy.
Vaso-occlusive crises (VOC) are the main clinical complication. They result from polymerization of HbS, deforming red blood cells and causing capillary occlusion, tissue hypoxia, intense bone pain, and frequent hospitalizations. In France in 2015, 25,150 hospitalizations were recorded, 61% of which were for VOC.
Iloprost is a prostacyclin (PGI2) analogue with vasodilatory, anti-platelet, anti-inflammatory, and antioxidant properties. It is used to treat severe limb ischemia and Raynaud's phenomenon, administered by IV infusion for 5 to 28 days.
It is well tolerated and has shown efficacy for bone pain related to bone marrow edema. Its rapid and sustained action makes it an interesting candidate for VOC, which are comparable to ischemic-origin pain. To date, only one reported case of iloprost use for a VOC exists, showing rapid and lasting improvement.
This randomized, multicenter, double-blind, placebo-controlled clinical trial aims to evaluate the efficacy of iloprost in patients hospitalized for VOC, with the objective of reducing pain and opioid consumption.
This comprehensive approach could significantly improve VOC management.

Start Date01 Sep 2026

Sponsor / Collaborator-

NCT07450469

/ Not yet recruitingNot ApplicableIIT

High-Altitude Iloprost Feasibility Study for Treatment of Frostbite

The goal of this observational study is to learn whether intravenous iloprost treatment for severe frostbite (grades 3-4) is feasible and safe when delivered in pre-hospital, remote high-altitude settings.
The main questions it aims to answer are:
* Is it feasible to initiate and deliver guideline-based intravenous iloprost for grade 3-4 frostbite at high altitude and what logistical barriers arise?
* Does field-based initiation of iloprost increase tissue preservation and reduce amputations (compared to historical cases from similar settings)?
Participants receiving Iloprost as part of a frostbite treatment will be followed up after discharge as well as 6, 12 and 18 months to assess tissue preservation and long-term sequelae from the initial frostbite injury.

Start Date01 May 2026

Sponsor / Collaborator-

NCT07263958

/ Not yet recruitingNot ApplicableIIT

HOLLYWOOD: Hemodynamic Evaluation and Management of Pulmonary Hypertension With Inhaled Iloprost in Chronic Obstructive Pulmonary Disease

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

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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2,905

Literatures (Medical) associated with Iloprost

01 Mar 2026·WILDERNESS & ENVIRONMENTAL MEDICINE

Prehospital Frostbite Management With Iloprost: Case Reports of Two Helicopter Evacuations in Northern Canada

Article

Author: Dion, Pierre-Marc ; O'Dochartaigh, Domhnall ; Soucy, Ryan ; Gauthier, Josianne ; Poole, Alexander

This case report describes iloprost infusion initiated in the prehospital setting to treat frostbite. Two cases were managed under a structured helicopter emergency medical services (HEMS) protocol during an extreme cold weather endurance race in the Yukon Territory in northern Canada. These cases demonstrate that intravenous iloprost delivery is operationally feasible in the prehospital setting. Administration was initiated on site and continued during helicopter transport following field rewarming and was completed in the hospital without complication. In remote or austere settings, field administration of iloprost can lower the time to treatment of frostbite injuries and may reduce warm ischemia time.

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 Feb 2026·AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Methodological considerations in the iloprost for septic shock trial

Article

Author: Chu, Qinjun ; Yang, Guanyu

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 2	Germany	Bayer AG	01 Feb 2015
Pneumonia	Phase 2	Denmark	Thrombologic ApS	01 Jun 2012
Pneumonia	Phase 2	Finland	Thrombologic ApS	01 Jun 2012
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

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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	nqblggbfvu(flfigukasx) = izyhqrldkp oggjdfmtig (txroogxiof )	Negative	01 Jul 2025
				Placebo	nqblggbfvu(flfigukasx) = voifalpkmw oggjdfmtig (txroogxiof )
NCT04040322 (CTgov)	Phase 3	CREST Syndrome \| Raynaud Disease \| Scleroderma, Systemic	198	Placebo IV infusion (Placebo)	hretsyixml(gnelodbdif) = igpbjinfpw ppmilyvfax (hyeykjufza, gxtmaopgjg - pevqwppxux) View more	-	25 May 2025
				Iloprost (Iloprost Injection, for Intravenous Use)	hretsyixml(gnelodbdif) = chzcjuetsl ppmilyvfax (hyeykjufza, elxtngrcsd - jgksnlbgxy) View more
NCT03867097 (CTgov)	Phase 2	CREST Syndrome \| Raynaud Disease \| Scleroderma, Systemic	34	Placebo IV infusion (Placebo)	uwhyeeocbb(etbqytsuha) = cwzsfpgfdc vvdxqknwyy (abjhcsgwmy, yitfavlvir - aoovqdvtly) View more	-	25 May 2025
				Iloprost (Iloprost Injection, for Intravenous Use)	uwhyeeocbb(etbqytsuha) = dbyecogqmj vvdxqknwyy (abjhcsgwmy, sywgwwuwvz - djbostdvxw) View more
NCT03044314 (OVATION, CTgov)	Phase 4	Hypertension, Pulmonary	27	nitric oxide+Iloprost	ilpypgkueu(vsbkuczqzf) = ctlddszpln oyrrjwzyuf (kaxageaqzb, vzosoubmpv - gtpnjmpsdv) View more	-	02 Apr 2024
NCT04598191 (Pubmed \| Can Anaesth Soc J)	Not Applicable	-	100	Inhaled iloprost (20 μg)	dazxdgbqdh(wvluqtvfop) = rdugsbxvaq hokrddaaxo (jqvhxzddjm, 3)	-	01 Apr 2024
				Saline	dazxdgbqdh(wvluqtvfop) = cjnjpfauwa hokrddaaxo (jqvhxzddjm, 3)
FDA Manual	Not Applicable	Frostbite	47	buflomedil+aspirin+buflomedil (Groups A)	dbxfwbqewk(fttoglcmht) = jnudntlwqo kvjzeeyuqh (mmyodmxyia )	Positive	13 Feb 2024
				iloprost+aspirin+buflomedil (Groups B)	dbxfwbqewk(fttoglcmht) = yrmglyzokp kvjzeeyuqh (mmyodmxyia )
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	cbynyzuryd(waqhrvinhs) = dupwzhytmw kjbpeqmuzx (gyhkofzjnz )	Positive	30 Aug 2023
ATS2023	Not Applicable	Hypertension, Pulmonary	-	Inhaled Iloprost	fhyfcgaoxz(acrqcnevow) = wgzsoxycva vrqejnhvvy (xvpzzddota, 12.2)	-	21 May 2023
				Inhaled Nitric Oxide	fhyfcgaoxz(acrqcnevow) = njbptcytib vrqejnhvvy (xvpzzddota, 12.2)
NCT03293407 (VENTASTEP, Pubmed \| J Med Internet Res)	Not Applicable	Pulmonary Arterial Hypertension	31	Inhaled Iloprost	dmcgslmgwq(wwvojgxcnk) = uivxnficwx xovqkolxop (wwbhuawbhy ) View more	-	08 Oct 2021
NCT00927654 (ILOCARD, Pubmed \| Can Anaesth Soc J)	Phase 3	-	253	Iloprost	enahiwzycf(degiusvgct) = iztqnlctad vdugrkcpae (cwikxlbrzj )	Negative	01 Aug 2019
				Placebo	enahiwzycf(degiusvgct) = lexoygzbja vdugrkcpae (cwikxlbrzj )