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Last update 05 Jun 2025

Eflornithine Hydrochloride

Last update 05 Jun 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(RS)-2,5-diamino-2-(difluoromethyl)pentanoic acid, 2-(difluoromethyl)ornithine, alpha-difluoromethylornithine

+ [18]

Target-

Action-

Mechanism-

Therapeutic Areas

NeoplasmsSkin and Musculoskeletal DiseasesOther Diseases+ [8]

Active Indication

High Risk NeuroblastomaHirsutismAdenomatous Polyposis Coli+ [11]

Inactive Indication

Facial HypertrichosisNeuroblastoma recurrent

Originator Organization

Sanofi

Active Organization

Almirall SA

Panbela Therapeutics, Inc.

Cancer Prevention Pharmaceuticals, Inc.

+ [5]

Inactive Organization

AbbVie, Inc.

Indiana University

Sanofi-Aventis U.S. LLC

License Organization

Michigan State University

MDD US Operations LLC

Orbus Therapeutics, Inc.

+ [2]

Drug Highest PhaseApproved

First Approval Date

United States (28 Nov 1990),

Facial Hypertrichosis

RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Australia)

Structure/Sequence

Molecular FormulaC6H15ClF2N2O3

InChIKeyFJPAMFNRCFEGSD-UHFFFAOYSA-N

CAS Registry96020-91-6

Clinical Trials associated with Eflornithine Hydrochloride

NCT06465199

/ Not yet recruitingPhase 1/2IIT

A Dose Escalation Study Using Difluoromethylornithine (DFMO) and AMXT-1501 Followed by a Randomized Controlled Trial of DFMO With or Without AMXT-1501 for Neuroblastoma, CNS Tumors, and Sarcomas

The purpose of this study is to evaluate the investigational drug AMXT 1501 (a pill taken by mouth) in combination with the drug difluoromethylornithine (DFMO) for infusion administered intravenously (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins). An investigational drug is one that has not been approved by the U.S. Food & Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.
The goals of this part of the study are:
* Establish a recommended dose of AMXT 1501 in combination with DFMO for infusion
* Test the safety and tolerability of AMXT 1501 in combination with DFMO for infusion in patients with cancer
* To determine the activity of study treatments chosen based on:
* How each subject responds to the study treatment
* How long a subject lives without their disease returning/progressing

Start Date01 Oct 2025

Sponsor / Collaborator

Hershey Medical Center

[+2]

NCT06976424

/ RecruitingPhase 4IIT

Comparison of Eflornithine Cream Combined With Intense Pulsed Light Versus Intense Pulsed Light Alone for Hirsutism in Women

The study compares the effects of a cream alone and a combined effect of cream and laser for the treatment of excessive facial hairs in women .

Start Date15 May 2025

Sponsor / Collaborator

Khyber Teaching Hospital

NCT06219174

/ RecruitingPhase 1/2IIT

Targeting ODC as an Immunotherapeutic Target in STK11 (LKB1) Pathway-Deficient NSCLC

The purpose of this study is to establish the safety, toxicity, and tolerability of Difluoromethylornithine (DFMO) in combination with pembrolizumab in advanced/metastatic Non-Small Cell Lung Cancer (NSCLC). Researchers also want to investigate how effective DFMO is at treating patients with advanced/ metastatic NSCLC.

Start Date07 Aug 2024

Sponsor / Collaborator

H. Lee Moffitt Cancer Center & Research Institute, Inc.

100 Clinical Results associated with Eflornithine Hydrochloride

100 Translational Medicine associated with Eflornithine Hydrochloride

100 Patents (Medical) associated with Eflornithine Hydrochloride

3,330

Literatures (Medical) associated with Eflornithine Hydrochloride

15 Jun 2025·INTERNATIONAL JOURNAL OF CANCER

Polyamine depletion limits progression of acute leukaemia

Article

Author: Verhelst, Steven H. L. ; Lock, Richard B. ; Mayoh, Chelsea ; Norris, Murray D. ; Ronca, Emma ; Spurling, Dayna ; Xiao, Lin ; Tanaka, Katsunori ; Somers, Klaartje ; Azfar, Mujahid ; Guo, Xinyi ; Cheung, Laurence C. ; Haber, Michelle ; Gao, Weiman ; Burns, Mark R. ; Karsa, Mawar ; Bongers, Angelika ; Kotecha, Rishi S. ; Vangheluwe, Peter ; Karsa, Ayu

Abstract:

Cancer cells are addicted to polyamines, polycations essential for cellular function. While dual targeting of cellular polyamine biosynthesis and polyamine uptake is under clinical investigation in solid cancers, preclinical and clinical studies into its potential in haematological malignancies are lacking. Here we investigated the preclinical efficacy of polyamine depletion in acute leukaemia. The polyamine biosynthesis inhibitor difluoromethylornithine (DFMO) inhibited growth of a molecularly diverse panel of acute leukaemia cell lines, while non‐malignant cells were unaffected. Responsiveness to DFMO was linked to decreased levels of its molecular target, the rate‐limiting polyamine biosynthesis enzyme ODC1, and of the polyamine transporters ATP13A2 and ATP13A3. DFMO increased polyamine uptake and upregulated expression of polyamine transporters in acute leukaemia cells, a compensatory effect abolished by treatment with the polyamine transport inhibitor AMXT 1501. This drug, currently in a phase 1 clinical trial in solid tumours in combination with DFMO, potentiated the inhibitory effects of DFMO, and their combination synergistically inhibited the growth of acute leukaemia cell lines by inducing apoptosis. DFMO and AMXT 1501 limited disease progression in highly aggressive xenograft models of infant KMT2A‐rearranged leukaemia, even when treatment was initiated at high disease burden. Increased expression of c‐MYC was associated with enhanced sensitivity to the combination of DFMO and AMXT 1501, suggesting this oncoprotein as a potential predictive marker of response to the drug combination. In conclusion, targeting polyamine biosynthesis and polyamine uptake limits disease progression in models of acute leukaemia, supporting further preclinical and clinical investigation into this approach for acute leukaemia.

20 May 2025·Nan fang yi ke da xue xue bao = Journal of Southern Medical University

[Spermine suppresses GBP5-mediated NLRP3 inflammasome activation in macrophages to relieve vital organ injuries in neonatal mice with enterovirus 71 infection].

Article

Author: Cao, Hong ; Chen, Xin ; Zhong, Baimao ; Zhang, Fangfang ; Tian, Zhihua ; Yang, Qingqing

OBJECTIVES:

To observe the therapeutic effect of spermine in neonatal mouse models of severe hand, foot and mouth disease (HFMD) caused by enterovirus 71 (EV71) infection and explore its therapeutic mechanism in light of regulation of macrophage GBP5/NLRP3 inflammasome pathway.

METHODS:

Neonatal BALB/c mice (3-5 days old) were divided into control group, EV71 infection group and Spermine treatment group. The mice in the latter two groups received an intraperitoneal injection of 50 μL EV71 suspension (1×10⁶ TCID50 of EV71), followed 3 days later by intraperitoneal injection of 50 μL PBS or 100 μmol/L spermine. GBP5, NLRP3, CXCL10, and TNFSF10 expressions in heart, liver, lung and kidney tissues of the mice were detected using Western blotting and qPCR, and tissue pathologies and macrophage infiltration were assessed with HE staining and immunohistochemistry. In cultured THP-1 and RAW264.7 cells, the effects of EV71 infection, GBP5 siRNA transfection and treatment with spermine or eflornithine on GBP5, NLRP3, CXCL10, and TNFSF10 mRNA expressions were investigated using qPCR.

RESULTS:

In the neonatal mice, EV71 infection resulted in multiple organ damage, macrophage infiltration and activation of the GBP5/NLRP3 pathway, and spermine treatment significantly improved tissue injuries, reduced macrophage infiltration, and down-regulated the expressions of GBP5, NLRP3 and the inflammatory factors in the infected mice. In THP-1 and RAW264.7 cells, EV71 infection caused significant upregulation of GBP5, NLRP3, CXCL10, and TNFSF10 expressions, which were obviously lowered by spermine treatment. In THP-1 cells, treatment with eflornithine significantly suppressed the reduction of GBP5, NLRP3, CXCL10, and TNFSF10 expressions induced by GBP5 siRNA transfection.

CONCLUSIONS:

Spermine suppressed EV71 infection-induced inflammatory responses by inhibiting GBP5-mediated NLRP3 inflammasome activation, suggesting a new strategy for treatment of severe HFMD.

15 May 2025·CLINICAL CANCER RESEARCH

A Phase II Trial of Naxitamab plus Stepped-up Dosing of GM-CSF for Patients with High-Risk Neuroblastoma in First Complete Remission

Article

Author: Basu, Ellen M. ; Cheung, Nai-Kong V. ; Cheung, Irene Y. ; Kramer, Kim ; Modak, Shakeel ; Roberts, Stephen S. ; Kushner, Brian H. ; Mauguen, Audrey

Abstract:

Purpose::

Naxitamab is a humanized form of the murine anti-GD2 mAb 3F8. In an international trial, naxitamab + GM-CSF was effective against chemoresistant high-risk neuroblastoma (HR-NB), leading to approval by the FDA. We now report results with patients in first complete remission (CR).

Patients and Methods::

The primary objective of this phase II protocol 16-1643 (Clinicaltrials.gov NCT03033303) was to assess event-free survival of patients with HR-NB in first CR treated with naxitamab + GM-CSF plus isotretinoin. HR-NB was defined as MYCN-amplified disease (any age) or metastatic disease at age >18 months. Cycles of immunotherapy were administered monthly up to five cycles and comprised (i) subcutaneously administered priming doses of GM-CSF 250 μg/m2/day on days −4 to −0 (Wednesday–Sunday), followed by a step-up to 500 μg/m2/day on days +1 to +5 (Monday–Friday) and (ii) naxitamab infused intravenously (30–90”) on days +1, +3, and +5 (Monday–Wednesday–Friday, i.e., three doses/cycle). The dosage of naxitamab was 3 mg/kg/infusion (9 mg/kg/cycle, i.e., ∼270 mg/m2/cycle). The dosage of isotretinoin was 160 mg/m2/day started after cycle 2, ×14 days/course, and ×6 courses.

Results::

Fifty-nine patients with HR-NB (53 stage 4, 6 stage 3) were enrolled from February 2017 to July 2020. At 36 months, event-free/overall survival rates were 73%/93%, but 50 of 59 patients received after protocol treatment (vaccine and/or difluoromethylornithine). Six of 18 relapses were isolated in the central nervous system. Longer time from diagnosis to enrollment was a significantly adverse prognostic factor (P = 0.04). Twenty-one of 59 patients took no isotretinoin. Treatment was tolerable allowing outpatient administration.

Conclusions::

Naxitamab + GM-CSF is a good option to consolidate first CR of patients with HR-NB, including those who did not undergo autologous stem-cell transplantation. Efforts to prevent central nervous system relapse are warranted.

127

News (Medical) associated with Eflornithine Hydrochloride

17 Apr 2025

·www.prnewswire.com

Norgine welcomes TGA registration in Australia of IFINWIL® (eflornithine) for adults and children diagnosed with high-risk neuroblastoma (HRNB)[1]

IFINWIL®: now TGA registered in Australia Children diagnosed with neuroblastoma are on average just 2 years old2 The average survival rate of children with HRNB is only 50%2 UXBRIDGE, England, April 17, 2025 /PRNewswire/ -- Norgine is pleased to announce that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL® (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy.1 Neuroblastoma Australia welcomed the news today: "On behalf of all families of children impacted by neuroblastoma, we welcome the TGA's decision to approve IFINWIL®. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers." said Lucy Jones, CEO Neuroblastoma Australia. High Risk Neuroblastoma is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life2. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high risk neuroblastoma3,4. Neuroblastoma originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases, it has spread by the time it is diagnosed5. "We are committed to improving the lives of children and their families living with high-risk neuroblastoma" said Gus Rudolph, General Manager, Norgine, Australia. "This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL® to patients as quickly as possible." "This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world," said Dr. David Gillen, Chief Medical Officer, Norgine. "By working collaboratively with international regulatory partners, we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We're proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines." Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL® is not currently included on the PBS. Please refer to the IFINWIL® Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing.1 Notes to Editors: About IFINWIL® IFINWIL® has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.6 IFINWIL® is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development 7. For more information on IFINWIL®, find the CMI here: IFINWIL Consumer Medicine Information (CMI) or the Therapeutic Goods Administration at or speak to your healthcare practitioner. High-Risk Neuroblastoma (HRNB) Treatment Background Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.8 Although there have been some improvements in survival, children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance, and have an extremely poor prognosis and low likelihood of long term survival9 (e.g. estimates as low as 15% of patients will live for five years after relapsing).10 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States11. About Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References Australian Product Information, IFINWIL®: IFINWIL Product Information (PI). Accessed 16.04.25 . Accessed 26.03.25 Australian Childhood Cancer Registry, Cancer Council Queensland. Australian Childhood Cancer Statistics Online (1983-2021). CCQ: Brisbane, Australia; October 2024. ANZCHOG-Position-Statement-Eflornithine-DFMO-use-for-patients-with-high-risk-neuroblastoma-in-Australia-and-New-Zealand-v2.0-23.08.2024.pdf . Accessed 16.03.25 Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov. Meyskens FL Jr, Gerner EW. Clin Cancer Res. 1999 May;5(5):945-51. 4. Sholler GLS, et al. Sci Rep. 2018 Sep 27;8(1):14445. Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57. Desai A, et al. J Clin Oncol. 2022 Jul; JCO2102478. 2. Lewis EC, et al. Int J Cancer. 2020 Dec;147(11):3152-3159 Bagatell R, DuBois SG, Naranjo A, et al. Children's Oncology Group's 2023 blueprint London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23. FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA, Accessed 02.04.25 Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalAccelerated Approval

15 Apr 2025

·www.pharmaceutical-business-review.com

Norgine agrees to acquire Theravia

Norgine has entered into a definitive agreement to acquire Theravia, a pharma laboratory that specialises in rare and debilitating condition treatments. The acquisition is expected to be a primary growth driver for Norgine in the medium to long term. Credit: National Cancer Institute on Unsplash. This transaction is said to be contingent on receiving customary regulatory approvals. Theravia’s portfolio includes Siklos for sickle cell disease and Orphacol for genetic disorders affecting the liver’s bile production. Both products are intended for the adult and paediatric population with these conditions. The products that encompass the therapeutic areas of rare haematology and hepatology are said to align with Norgine’s current portfolio of rare and specialty medicines. With the addition of Theravia’s offerings, Norgine’s rare disease portfolio will now feature six core products: eflornithine, mavorixafor, Pedmarqsi, Agilus, Orphacol, and Siklos. This acquisition is expected to be a primary growth driver for Norgine in the medium to long term. Recent transactions involving Fennec Pharma’s Pedmarqsi and X4 Pharma’s mavorixafor highlight Norgine’s focus on expanding its presence in the rare disease and specialty pharmaceuticals market by means of acquisitions as well as in-licensing. Norgine CEO Janneke van der Kamp said: “This acquisition is a unique opportunity for Norgine to bolster our growth trajectory, as well as our rare disease portfolio. “With our strong legacy and proven track record of successfully bringing innovative treatments to patients, we believe we are well placed to ensure the Theravia medicines reach their full potential.” Theravia was established via the Addmedica and CTRS merger, with an aim to fulfil the medical requirements of individuals suffering from these difficult-to-treat ailments. Theravia CEO Franck Hamalian said: “The joining of our companies brings together the skills, know-how and geographical presence that will create a European-based champion of rare and ultra rare disease. “Our combined product portfolios and Norgine’s legacy and experience will enable broader access to medicines for patients with high unmet medical needs.

Acquisition

15 Apr 2025

·www.prnewswire.com

Norgine strengthens rare disease portfolio with acquisition of Theravia

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines. Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe. PARIS, April 15, 2025 /PRNewswire/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions. Recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing. Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS®, for adults and children with sickle cell disease, and ORPHACOL®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France. said Janneke van der Kamp, Chief Executive Officer, Norgine. Franck Hamalian, Chief Executive Officer, Theravia, added: With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS® and ORPHACOL®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term. Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth. The transaction remains subject to obtaining customary regulatory approvals from the competent authorities.

Drug ApprovalAcquisitionOrphan DrugPhase 3

100 Deals associated with Eflornithine Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Eflornithine Hydrochloride	P01CX03 D11AX16	DB06243

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
High Risk Neuroblastoma	United States	MDD US Operations LLC	13 Dec 2023
Hirsutism	European Union	Almirall SA	19 Mar 2001
Hirsutism	Iceland	Almirall SA	19 Mar 2001
Hirsutism	Liechtenstein	Almirall SA	19 Mar 2001
Hirsutism	Norway	Almirall SA	19 Mar 2001
Facial Hypertrichosis	United States	Sanofi-Aventis U.S. LLC	28 Nov 1990

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Astrocytoma	Phase 3	United States	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	Belgium	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	Canada	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	France	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	Germany	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	Italy	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	Netherlands	Orbus Therapeutics, Inc.	01 Jul 2016
Astrocytoma	Phase 3	United Kingdom	Orbus Therapeutics, Inc.	01 Jul 2016
Glioblastoma	Phase 3	United States	Orbus Therapeutics, Inc.	01 Jul 2016
Glioblastoma	Phase 3	Belgium	Orbus Therapeutics, Inc.	01 Jul 2016

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03794349 (ANBL1821, ASCO2025)	Phase 2	Refractory Neuroblastoma MYCN	91	DIT	ctcmbfoczh(rydsgeqhow) = 55.6% (5/9) of Arm B pts developed hearing loss requiring DFMO dose hold. With discontinuous dosing, hearing loss was 15.8% (6/38) on Arm B compared to 6.3% (2/32) on Arm A ohykcaqpkk (ukadpsttta )	Negative	30 May 2025
				DIT with DFMO
NCT02794428 (CTgov)	Phase 2	Carcinogenesis \| Gastritis, Hypertrophic \| Stomach Cancer	91	Eflornithine (Eflornithine)	jweticepxw(voneeviwds) = brwkevtzct saaywtceas (nmhklnvhal, 21.05) View more	-	05 Apr 2024
				Eflornithine placebo (Eflornithine Placebo)	jweticepxw(voneeviwds) = sdftpkccvg saaywtceas (nmhklnvhal, 21.60) View more
NCT03794349 (Literature \| Pubmed \| Br J Cancer) Manual	Phase 1	Neuroblastoma recurrent	24	DFMO+celecoxib+cyclophosphamide +topotecan	lbakubquah(ibqqofwqdk) = bljjjzlzbt bsikwmmjiu (uxzigktxpu ) View more	Positive	10 Jan 2024
NCT02395666 (NMTRC003B, Pubmed \| J Clin Oncol)	Phase 2	High Risk Neuroblastoma Maintenance	141	Eflornithine (DFMO)	sjtgfbmppt(bvyzsolksi): HR = 0.5 (95% CI, 0.29 - 0.84)	Positive	01 Jan 2024
				No-DFMO/control group
NCT02395666 (ANBL0032, FDA) Manual	Phase 2	High Risk Neuroblastoma	360	IWILFIN	pcnldhmwgr(uhxkfudkee): HR = 0.48 (95% CI, 0.27 - 0.85) View more	Positive	13 Dec 2023
				control
NCT02395666 \| NCT00026312 (ASCO2022)	Phase 2	High Risk Neuroblastoma Maintenance	140	DFMO	hvkfonrcpt(hjfialjbby): hazard ratio = 0.48 (95% CI, 0.27 - 0.85), P-Value = 0.0114 View more	Positive	02 Jun 2022
				eflornithine (DFMO) (Control)
NCT01483144 (Pubmed \| N Engl J Med)	Phase 3	Adenomatous Polyposis Coli	171	Eflornithine	-	-	10 Sep 2020
				Sulindac
NCT02395666 (NMTRC003/003b, Pubmed \| Int J Cancer)	Phase 2	High Risk Neuroblastoma Maintenance	81	DFMO	xinfwobpft(unsynkucof) = vsnylmtumo geubsfcixj (lluktkqner, 79.3% - 94.2%) View more	Positive	11 May 2020
				Standard upfront therapy	xinfwobpft(unsynkucof) = awabdnxzwh geubsfcixj (lluktkqner, 69.5% - 88.3%) View more
NCT02030964 (N2012-01 \| NANT 2012-01, ASCO2018)	Phase 1	High Risk Neuroblastoma	24	DFMO and celecoxib with Cyclo/Topo	rhgynpclho(ysmqlofddm) = There were three cycle-1 DLTs (hematologic; anorexia; transaminitis) and two DLTs in later cycles (cycle-2 hematuria; cycle-11 hypotension) lqjmjnakzs (apklgxpjys ) View more	-	01 Jun 2018
NCT00006101 (CTgov)	Phase 2	Prostate Cancer, Familial	76	eflornithine (Eflornithine)	kukrefxdtp(rfdtqdvzag) = rhlaqdbjni ndjrorgxpt (lvvftzuipq, 26.29) View more	-	12 Apr 2018
				Placebo (Placebo)	kukrefxdtp(rfdtqdvzag) = rzjhosmlcw ndjrorgxpt (lvvftzuipq, 38.29) View more

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