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Last update 22 Jan 2025

Darvadstrocel ( TiGenix)

Last update 22 Jan 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Mesenchymal stem cell therapy

Synonyms

Darvadstrocel, CX601, Alofisel

+ [1]

Target-

Mechanism

Cell replacements

Therapeutic Areas

Digestive System DisordersOther Diseases

Active Indication

Perianal fistulaRectal FistulaPerianal fistula due to Crohn's disease

Inactive Indication

Colitis, UlcerativeCrohn DiseaseRectovaginal Fistula

Originator Organization

TiGenix SAU

Active Organization

Tigenix SAU

Takeda Pharmaceutical Co., Ltd.

TiGenix NV

+ [1]

Inactive Organization

Hospital Universitario La Paz

Takeda Canada, Inc.

Drug Highest PhaseApproved

First Approval Date

EU (23 Mar 2018),

Perianal fistula

Regulation-

Clinical Trials associated with Darvadstrocel ( TiGenix)

NCT05974280

/ RecruitingNot ApplicableIIT

Pilot Study on the Treatment of Anal Fistulas in Crohn's Disease Patients Using Alofisel Versus Fat Autologous Stem Cells

One of the newest and most innovative medicinal approaches is cell therapy. Several clinical trials and experimental investigations have looked into the feasibility of treating CD-related fistulas with stem cells.
The current indication for ALOFISEL® (active ingredient: Darvadstrocel) is the treatment of difficult perianal Crohn's fistulas that have not responded well to at least one conventional therapy or biotherapy.
This brand-new cell therapy medication is created using amplified allogeneic human adult mesenchymal stem cells from adipose tissue (ADSC). The supplier mandates that two patients be booked for a single dose of ALOFISEL® due to the medication's expensive price-roughly €54,000 for a single dose of 120 million-which cannot be stored once thawed.
Only one of the two patients receives therapy; the other serves as the backup patient. By doing this, another "back-up" patient who might receive no care at all is avoided.
An developing alternate approach to allogeneic ADSC injection for the treatment of complicated anal fistulas in CD is autologous fat injection. In recent years, autologous fat grafts have been the subject of in-depth research. They are popular because it is simple to get clinical samples (lipoaspirate, adipose tissue), and because there are a lot of ADSCs in adipose tissue. Additionally, ADSCs show strong immunomodulatory and regenerative capacities.
We would wish to compare the effectiveness of these two injection kinds on perianal fistulas as part of our care of CD.

Start Date02 Jan 2024

Sponsor / Collaborator

Nantes University Hospital

NCT05113095

/ RecruitingNot Applicable

Postmarketing Surveillance Registry to Evaluate Safety and Efficacy of Darvadstrocel for the Treatment of Complex Perianal Fistulas in Crohn's Disease Patients (J-INSPIRE) (All-case Surveillance)

This study is a survey in Japan of Darvadstrocel injection used to treat Crohn's disease people with complicated anal fistula. The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.
The main aim of the study is to check for side effects related from Darvadstrocel injection and to check if Darvadstrocel injection improves symptoms of Crohn's disease. During the study, participants with Crohn's disease will take Darvadstrocel injection according to their clinic's standard practice. The study doctors will check for side effects from Darvadstrocel for 36 months.

Start Date30 Nov 2021

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

NCT04701411

/ RecruitingPhase 3

A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Darvadstrocel in the Treatment of Complex Perianal Fistula in Pediatric Subjects With Crohn's Disease Over a Period of 24 Weeks and an Extended Follow-up Period for a Total of up to 52 Weeks

A perianal fistula is an abnormal passageway that develops between the rectum and the skin near the anus. The fistula is considered complex if it branches into several openings or an abscess is also present.
The main aim of this study is to learn if complex perianal fistulas in children and teenagers close after treatment with darvadstrocel.
2 to 3 weeks before treatment with darvadstrocel, each participant will have surgery to clean the fistula and to drain any abscesses. On the day of treatment, each participant will have the fistula cleaned and will receive an injection of darvadstrocel near the fistula, under anesthetic.
For up to 1 year after treatment, participants will regularly visit the clinic for follow-up. The fistula will be examined and any side effects from the treatment will be recorded. Participants will have an MRI at one clinic visit (about 24 weeks after treatment).

Start Date30 Jun 2021

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

[+1]

100 Clinical Results associated with Darvadstrocel ( TiGenix)

100 Translational Medicine associated with Darvadstrocel ( TiGenix)

100 Patents (Medical) associated with Darvadstrocel ( TiGenix)

Literatures (Medical) associated with Darvadstrocel ( TiGenix)

01 Nov 2024·Clinics and Research in Hepatology and Gastroenterology

Real-life effectiveness of allogeneic expanded adipose-derived mesenchymal stem cell therapy for perianal fistulizing refractory Crohn's disease

Article

Author: Altwegg, Romain ; Cotte, Eddy ; Nancey, Stéphane ; Danion, Pauline ; Boschetti, Gilles ; Charlois, Anne Laure ; Alonso-Gomez, Maria ; Gay, Claire ; Malezieux, Emilie

BACKGROUND:

Real life data about the long-term efficacy of darvadstrocel for treating perianal fistulas in Crohn's disease (CD) remain scarce.

AIM:

To report the effectiveness and safety of darvadstrocel therapy to close perianal fistula in a real-life cohort of CD patients.

METHODS:

All patients with CD suffering complex draining perianal fistulas who consecutively underwent administration of a single local injection of darvadstrocel at two centres were followed up and evaluated. The primary endpoint was clinical remission (closure of all external openings with no discharge at pressure) at week 24. Combined remission (defined as clinically plus MRI-assessed fistula closure) were also assessed at week 24 and 52.

RESULTS:

A total of 36 CD patients (19 Male, mean age 38.5 years) were included with a median follow up of 16 months. Clinical remission was achieved in 17 out of 36 patients (47.2 %) and combined remission in 15 out of 36 patients (41.6 %) at week 24. At week 52, clinical and combined remission was achieved in 17 out of 36 patients (47.2 %) and in 15 out of 33 evaluable patients (45.4 %), respectively.

CONCLUSION:

In this real-world setting, a successful response to darvadstrocel therapy based on clinical remission was reported in around half of the patients and combined remission including radiological assessment in more than 4 out of 10 patients.

02 Aug 2024·EXPERT OPINION ON BIOLOGICAL THERAPY

The potential for medical therapies to address fistulizing Crohn’s disease: a state-of-the-art review

Review

Author: Lakatos, Peter L. ; Bessissow, Talat ; Shehab, Mohammad ; De Marco, Davide

INTRODUCTION:

Crohn's disease (CD) is a chronic, relapsing immune mediated disease, which is one of the two major types of inflammatory bowel disease (IBD). Fistulizing CD poses a significant clinical challenge for physicians. Effective management of CD requires a multidisciplinary approach, involving a gastroenterologist and a GI surgeon while tailoring treatment to each patient's unique risk factors, clinical representations, and preferences.

AREAS COVERED:

This comprehensive review explores the intricacies of fistulizing CD including its manifestations, types, impact on quality of life, management strategies, and novel therapies under investigation.

EXPERT OPINION:

Antibiotics are often used as first-line therapy to treat symptoms. Biologics that selectively target TNF-α, such infliximab (IFX), have shown high efficacy in randomized controlled trials. However, more than 50% of patients lose response to IFX, prompting them to explore alternative strategies. Current options include adalimumab and certolizumab pegol combination therapies, as well as small-molecule drugs targeting Janus kinases such as Upadacitinib. Furthermore, a promising treatment for complex fistulas is mesenchymal stem cells such as Darvadstrocel (Alofisel), an allogeneic stem cell-based therapy. However, surgical interventions are necessary for complex cases or intra-abdominal complications. Setons and LIFT procedures are the most common surgical options.

01 May 2024·Wiener klinische Wochenschrift

Efficacy of cx601 (darvadstrocel) for the treatment of perianal fistulizing Crohn’s disease—A prospective nationwide multicenter cohort study

Article

Author: Riss, Stefan ; Harpain, Felix ; Czipin, Sascha ; Pramhas, Michael ; Scharitzer, Martina ; Dawoud, Christopher ; Stift, Anton ; Widmann, Kerstin Melanie

Summary:

Background:

The use of mesenchymal stem cells is considered a novel and promising therapeutic option for patients with perianal fistulizing Crohn’s disease; however, data on its clinical application remain scarce. This multicenter nationwide study aimed to assess the clinical efficacy of mesenchymal stem cells in closing complex anal fistulas.

Methods:

In this study 14 Crohn’s disease patients (3 males, 11 females) with complex anal fistulas treated in 3 tertiary hospitals in Austria were included between October 2018 and April 2021.Injection of 120 million allogeneic expanded adipose-derived mesenchymal stem cells (Cx601—darvadstrocel) was performed in each patient. Closure of the external fistula opening without secretion by external manual compression was defined as treatment success.

Results:

The median age of the patient population at the time of surgery was 32 years (range 26–53 years) with a median body mass index of 21.7 kg/m2 (range 16.7–26.6 kg/m2). Of the patients 12 (86%) received monoclonal antibodies (infliximab, adalimumab, ustekinumab, vedolizumab) at the time of surgery.The median number of complex fistulas was 1.4 (range 1–2), The median operative time was 20 min (range 6–50 min) with no perioperative complications.After a median follow-up of 92 weeks, we found successful fistula closure in 57.1% (n = 8) of treated patients. The perianal disease activity index did not improve significantly from initially 7 to a median of 6 after 52 weeks (p = 0.495).

Conclusion:

Darvadstrocel is a safe, minimally invasive surgical technique without significant perioperative complications. Clinical success can be expected in about half of the treated patients.

News (Medical) associated with Darvadstrocel ( TiGenix)

10 Jan 2025

·www.businesswire.com

Clinical and Commercial Developments in MSCs: 1,670+ Trials Highlight Versatile Applications and Emerging Opportunities - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "Mesenchymal Stem Cells / Medicinal Signaling Cells (MSCs) - Advances & Applications, 2025" report has been added to ResearchAndMarkets.com's offering. MSCs are multipotent cells that have been referred to as mesenchymal stem cells, medicinal signaling cells, and mesenchymal stromal cells, reflecting their versatility and diverse applications. MSCs can differentiate into a variety of cell types, such as osteoblasts, chondrocytes, myocytes, and adipocytes. Their unique ability to secrete factors that promote tissue repair and modulate their microenvironment with anti-inflammatory and anti-fibrotic effects makes them essential in regenerative medicine. Furthermore, their immuno-evasive properties make MSCs particularly suitable for allogenic transplantation. MSCs serve as vital raw materials across numerous industries, including regenerative medicine, cosmeceuticals, and cultured meat production. Key applications include: Cell-based therapies for musculoskeletal diseases, spinal injuries, and cardiovascular conditions. Engineered tissues and organs, such as skin, bone, blood vessels, and liver. Cell-derived products, such as exosomes and growth factors, utilized in cosmetics and skincare. Cultured meat production, leveraging MSCs' role in muscle development and their ability to differentiate into muscle and fat cells. Globally, 12 MSC-based therapies have been approved for various indications, with the majority concentrated in South Korea, Japan, and the EU. Specifically, the Republic of Korea has approved five products: Queencell from Anterogen, Cellgram AMI from Pharmicell, Cupistem from Anterogen, Cartistem from Medipost, and NeuroNataR from Corestem. Japan has approved two products: Temcell HS from JCR Pharmaceuticals and Stemirac from Nipro Corporation. The EMA in Europe has approved two products: Holoclar from Chiesi Farmaceutici and Alofisel from TiGenix/Takeda. India has approved one product: Stempeucel from Stempeutics. Iran has approved one product: MesestroCell developed by Cell Tech Pharmed. Finally, Australia has approved one product: Remestemcel-L from Mesoblast. Despite this progress, no MSC-based therapeutic have yet received U.S. FDA approval, although the FDA is actively reviewing Mesoblast's Remestemcel-L. The cosmeceutical market is also rapidly adopting MSC-derived products, particularly exosomes, to leverage their anti-aging and regenerative properties, with companies like L'Oreal and Johnson & Johnson investing heavily in this space. Similarly, cultured meat companies are utilizing MSCs for producing muscle and fat cells, enhancing taste and texture. Groundbreaking approvals, such as lab-grown chicken by Upside Foods and Good Meat, have paved the way for further growth in this industry. MSC-based gene editing is expanding, enabling the overexpression of antitumor genes and therapeutic factors. Researchers are also exploring the use of MSCs in 3D printing and engineered organ production, with breakthroughs anticipated in the next decade. Importantly, companies like Cynata Therapeutics are pioneering iPSC-derived MSC production technologies, enabling large-scale therapeutic development. Today there are at least eight companies who are involved with the development of iPSC-derived MSCs therapeutics (iMSCs), including Cynata Therapeutics, Eterna Therapeutics, Implant Therapeutics, Bone Therapeutics, Brooklyn ImmunoTherapeutics, Fujifilm CDI, Citius Pharmaceuticals, and Kiji Therapeutics. There are also over 1,670 clinical trials involving MSCs are registered on ClinicalTrials.gov. Nearly 75% of these clinical studies are using MSCs for the development of regenerative medicine (RM) products. Approximately 14% of the studies are using MSCs for disease modeling. The remaining 11% of the studies are using MSCs for drug discovery and cytotoxicity testing applications. Challenges in these trials include maintaining MSC phenotype and functionality during expansion. However, ongoing advancements suggest a promising future, with projections for at least 50 globally approved MSC-based products by 2040, averaging 3-4 new approvals annually. Key Players in MSC Research Products The demand for MSC-based research products has surged in recent years, with major suppliers expanding their portfolios. Companies like RoosterBio have increased the access to quality MSCs through supply chain industrialization, standardized cell bank product forms, and fit-for-purpose cGMP-compatible cells and media systems. Other leading MSC research product suppliers include PromoCell, Lonza, Thermo Fisher Scientific, STEMCELL Technologies, Miltenyi Biotec, Bio-Techne (R&D Systems), ScienCell Research Laboratories, and the ATCC. Challenges and Opportunities While MSC commercialization faces hurdles-such as scalability and regulatory approval-opportunities abound. The market for MSC-based products is poised for notable growth across the research, therapeutic, cosmetic, and food industries. Within this rapidly changing landscape, having a thorough understanding of the competition is essential. This global strategic report presents rates of MSC clinical trials, scientific publications, patents, funding events, and collaborations. It presents the pipeline for MSC-based therapeutics, identifies market opportunities and threats, and explores future directions and emerging applications. It reveals innovative MSC products, services, and technologies offered by 125 leading competitors. Finally, it presents detailed market size figures for the global MSC market, segmented by geography and business segment with future forecasts through 2031. Key Topics Covered: MESENCHYMAL STEM CELLS (MSCs): AN OVERVIEW Stabilization of hMSCs as a Technology The Impact of MSCs on Regenerative Medicine Timeline of MSC Nomenclature Sources of MSCs Cell Surface Markers in MSCs In vitro Differentiation Potentials of MSCs Soluble Factors Secreted by MSCs Stemness Genes Present in MSCs Types of MSCs and their Biomedical Applications Genes that are Pivotal for MSC's Properties MSC-BASED CLINICAL TRIAL LANDSCAPE MSC-Based Clinical Studies by Geography Sources of MSCs used in Current Clinical Trials Share of Autologous & Allogeneic MSCs Clinical Trials MSC-Based Clinical Trials by Disease Indication MSC-Based Clinical Trials by Phase of Study Select MSC-Based Clinical Trials MESENCHYMAL STEM CELLS (MSCS): PATENT LANDSCAPE MSC Patent Publications by Jurisdiction MSC Patent Applicants MSC Patent Inventors MSC Patent Owners Legal Status of MSC Patent Applications MSC-Derived Exosome Patents Patents for iPSC-Derived MSCs MSC Patents by Therapy Type PUBLISHED SCIENTIFIC PAPERS ON MSCS MSC MANUFACTURING PLATFORMS: AN OVERVIEW Methods of Isolations of MSCs Cell Expansion: Conventional Cultures to Bioreactors Downstream Processing of MSCs Autologous & Allogeneic MSC Manufacturing: A Comparison MSC Manufacturing Economics in Small Scale Large Scale Manufacturing of MSCs CELL & GENE THERAPY CONTRACT MANUFACTURING Staff Shortage Need for Automation Capacity Constraints CDMO Platforms with Greater Potentials CMOs/CDMOs Geographic Distribution of Manufacturing Facilities Continued Rise of Outsourcing Major CDMOs in the U.S Major Cell & Gene Therapy CDMOs in Europe U.K.'s Domination in European Contract Manufacturing BIOENGINEERING SOLUTIONS TO BOOST MSC'S FUNCTIONS Small Molecule Priming of MSCs Particle Engineering of MSCs Genetic Engineering of MSCs Examples of Bioengineered MSCs to Produce Anticancer Therapeutics Engineered MSCs for Neurological Conditions Engineered MSCs for Cardiovascular Diseases Engineered MSCs for Lung Injury Engineered MSCs for Diabetes Bioengineering Approaches to Improve MSC Administration MAJOR DISEASES ADDRESSED BY MSCS Studies Using MSCs for Autoimmune Diseases Studies using MSCs for Cardiovascular Diseases Studies using MSCs for Neurodegenerative Diseases Studies using MSCs for Bone & Cartilage Diseases Studies using MSCs in GvHD Studies using MSCs in Crohn's Disease Studies using MSCs in Type 1 Diabetes Studies using MSCs in Systemic Lupus Erythematosus (SLE) Studies using MSCs in Parkinson's Disease Studies using MSCs in Alzheimer's Disease Studies using MSCs in Kidney Failure Studies using MSCs in Spinal Cord Injury Studies using MSCs for Wound Healing RECENT COLLABORATIONS IN MSC SECTOR, 2020-2024 Collaboration between BioSolution Designs & RoosterBio Collaboration between CytoMed Therapeutics, Ltd. & Sengkang General Hospital Collaboration between REPROCELL & Histocell Collaboration between FUJIFILM Dyosynth & RoosterBio RoosterBio's Collaboration with AGC Biologics RoosterBio's Partnership with ShiftBio RoosterBio's Partnership with Univercells Technologies Collaboration between Cynata & Fujifilm Collaboration between American CryoStem Corp. & BioTherapeutic Lab Corp. RoosterBio's Collaboration with Sartorius Collaboration between American CryoStem and CRADA RoosterBio's Collaboration with Sartorius Korea Biotech Collaboration between Catalent & BrainStorm Collaboration between Cipla & Stempeutics Aethlon's Collaboration with University of Pittsburgh RoosterBio's Collaboration with Senti Biosciences MSC-BASED PRODUCTS IN THE MARKET Approved MSC Therapies Queencell Cellgram AMI Cupistem Cartistem NeuroNataR Holoclar Remestemcel-L Temcell HS Stempeucel Alifosel MesestroCell Stemirac Marketed Biomaterial Carrier-Based MSCs & MSC Progenitors Osteocel AlloStem Grafix Cellentra VCBM Trinity ELITE Map3 BIO4 Trinity Evolution Carticel Chondron DeNovo NT Chondrocelect Ossron JACC MACI Ortho-ACI Ossgrow Cartigrow Topical Cosmetic Products Containing MSCs/MSC-Derived Exosomes MARKET ANALYSIS Market Size for MSC Therapies Global Demand for Mesenchymal Stem Cells (MSCs) COMPANY PROFILES Advancells Aegle Therapeutics AGC Biologics AlloSource, Inc. Ambulero American CryoStem Corporation American Type Culture Collection (ATCC) Amniotics Andelyn Biosciences Anemocyte S.r.l Anterogen, Co., Ltd. Avid Bioservices, Inc. Bacthera Baylx, Inc BioCardia BioCentriq BioEden, Inc Bioinova s.r.o BioRestorative Therapies Bioscience Institute S.p.A Blue Horizon International Boehringer Ingelheim BioXellence Bonus Biogroup, Ltd BrainStorm Cell Therapeutics, Inc. Catalent CCRM Cell2Cure ApS Cell Care Therapeutics Cellcolabs CELLeBRAIN Cellipont Bioservices Celprogen, Inc CellProthera CellResearch Corporation, Pte, Ltd. Cell Surgical Network (CSN) Celltex Therapeutics Corporation CellTherapies Cellular Biomedicine Group Charless River Laboratories CHIESI Farmaceutici S.p.A Citius Pharmaceuticals, Inc CorestemChemon, Inc. Creative Bioarray Creative Medical Technology Holdings, Inc Curia Global, Inc Cynata Therapeutics, Ltd. Cytovance Biologics Defined Bioscience, Inc Direct Biologics eQcell, Inc. Excellos Exosla Therapeutics EXOSOMEplus Exothera Fujifilm Diosynth Biotechnologies Future Cell Japan, Co., Ltd. Genezen GenScript Biotech Corporation Hope Biosciences, Implant Therapeutics, Inc INCELL Corporation InGeneron Invitrx Therapeutics Japan Tissue Engineering, Co., Ltd. (J-TEC) JCR Pharmaceuticals, Co., Ltd. Jointechlabs Kangstem Biotech, Co., Ltd. Kimera Labs LifeCell Longeveron Lonza Group, Ltd Lorem Cytori USA, Inc. MEDPOST Mesoblast, Ltd Millipore Sigma NecstGen NextCell NIPRO Corporation Novadip Biosciences Novus Biologicals NuVasive OCT Therapies & Research Pvt., Ltd OmniaBio, Inc Orthocell, Ltd Orthofix Medical, Inc. Ossium Health Personal Cell Sciences Corporation Personalized Stem Cells, Inc. Pfizer CentreOne Pluri Biotech, Ltd. Porton Advanced Prometheus Life Technologies PromoCell PuREC, Co., Ltd Regrow Biosciences Reliance Life Sciences Remedy Cell Rentschler Biopharma REPROCELL USA Resilence RESTEM RHEACELL Richter-HELM RoosterBio RTI Surgical RoslinCT Samsung Biologics San Bio, Co., Ltd. Smith & Nephew, Inc StemBioSys, Inc STEMCELL Technologies, Inc Stemedica Cell Technologies, Inc Stemmatters StemMedical Stempeutics Research Pvt., Ltd StromaBio TaiwanBio Therapeutics, Co., Ltd Takeda Pharmaceutical, Co., Ltd Tempo Bioscience ThermoFisher Scientific Vericel Corporation Wuxi App Tec Xintela For more information about this report visit https://www.researchandmarkets.com/r/upnyhu About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Drug ApprovalClinical Study

16 Dec 2024

·pipelinereview.com

Takeda Provides an Update on Alofisel® (darvadstrocel)

OSAKA, Japan and CAMBRIDGE, MA, USA I December 13, 2024 I Takeda ( TSE:4502/NYSE:TAK ) today announced that the company is working with the European Medicines Agency (EMA) to voluntarily withdraw the marketing authorization of Alofisel® (darvadstrocel), a treatment for complex perianal fistulas in patients with Crohn’s disease, in the European Union (EU). Alofisel is an allogeneic stem cell therapy that is approved in the EU for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. 1 Alofisel should be used only after conditioning of the fistulas. The initial authorization of Alofisel in the EU was based on results from the Phase 3 ADMIRE-CD placebo-controlled registrational study. 1 Given the small population size (n = 212) and modest benefit observed in the ADMIRE-CD study (a difference of 15.8% between the modified intention-to-treat population versus placebo at 24 weeks), Takeda agreed to provide results from ADMIRE-CD II, a study that was underway, to confirm Alofisel’s efficacy. 1-3 In October 2023, Takeda communicated ADMIRE-CD II , a randomized placebo-controlled study of 568 patients with complex CPF, did not meet its primary endpoint of combined remission at 24 weeks. 3-5 Additional results, which were presented at the European Crohn’s and Colitis Organisation Congress 2024 in February, showed no statistical differences in any of the secondary endpoints. 5 The safety profile for Alofisel was consistent with prior studies as no new, emerging safety signals were identified. 2,4,5 “Guided by our values and the needs of patients living with this difficult-to-treat condition, we engaged the EMA, as well as health care professionals and others in the community, to review the totality of data and the role of our medicine,” said Ramona Sequeira, president, Global Portfolio Division, at Takeda. “Those valuable discussions indicated that despite the conflicting results across our data, the clinical benefit of Alofisel is no longer sufficient to justify its continued use in the EU. We recognize the difficulty of this outcome for patients and will work closely with the Crohn’s Perianal Fistulas (CPF) community during this transition.” In addition to the EU, Takeda is engaging health authorities about this outcome in countries where Alofisel is currently approved, keeping the best interest of patients at the forefront. Takeda is committed to continuing to share data from clinical and observational studies for the benefit of patients and the entire health care community, with the hope that these insights can contribute to scientific advancements in complex CPF. Health care professionals with medical-related questions about Alofisel should contact Takeda Medical Information at medinfoEMEA@takeda.com or medinfoAPAC@takeda.com . Patients should consult their treating health care professional if they have questions. About Alofisel® Alofisel® (darvadstrocel) is a dispersion for injection of expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (expanded adipose stem cells – eASC) for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease. Therapeutic Indications Alofisel is indicated for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. Alofisel should be used only after conditioning of the fistulas. Important Safety Information Contraindications Hypersensitivity to the active substance, bovine serum or to any of the excipients. Special warnings and special precautions for use Alofisel may contain trace amounts of either gentamicin or benzylpenicillin and streptomycin. This should be considered in patients with known hypersensitivity to these classes of antibiotics. Local anaesthesia is not recommended due to the unknown effect of local anaesthetics on the injected cells. The injection of any substance other than sodium chloride 9 mg/mL (0.9%) solution (e.g. hydrogen peroxide, methylene blue, iodine solutions or hypertonic glucose solutions) through the fistula tracts is not allowed before, during, or after the injection of Alofisel as these may compromise the viability of the cells. Alofisel must not be administered using a needle thinner than 22G. Thinner gauge needles can cause cell disruption during injection and may compromise cell viability and, therefore, may affect efficacy of treatment. As Alofisel is a living stem cell therapy it cannot be sterilised, a risk of transmission of infectious agents exists, although the risk is considered to be low and controlled in the manufacturing process. Healthcare professionals administering darvadstrocel must, therefore, monitor patients for signs and symptoms of infections after treatment and treat appropriately, if needed. Alofisel should only be administered by specialist physicians experienced in the diagnosis and treatment of conditions for which darvadstrocel is indicated. Patients treated with Alofisel must not donate blood, organs, tissues and cells for transplantation. The traceability requirements of cell-based therapy medicinal products must apply. Fertility, Pregnancy & Lactation No data is available from the use of darvadstrocel in pregnant women. Darvadstrocel is not recommended during pregnancy and in women of childbearing potential who are not using contraception. As a precautionary measure, darvadstrocel is not recommended for administration during breast-feeding. Undesirable effects Based on clinical trial and post-marketing data, the most commonly reported adverse drug reactions were anal abscess, proctalgia and anal fistula with the most commonly reported serious adverse drug reactions of anal abscess and anal fistula. Procedural pain is associated to conditioning reactions occurring up to seven days after the fistula preparation for treatment administration. For EU audiences, please see the Summary of Product Characteristics (SmPC) for Alofisel®. Please consult with your local regulatory agency for approved labeling in your country. Takeda in Gastroenterology Takeda is committed to meeting the very real needs of those living with gastrointestinal (GI) diseases. We believe that GI and liver diseases are life-disrupting conditions. Beyond a fundamental need for effective treatment options, we understand that improving patients’ lives also depends on their needs being recognized. With more than 35 years of experience in gastroenterology, Takeda has made significant strides in addressing patient needs with treatments for inflammatory bowel disease, eosinophilic esophagitis, acid-related diseases, short bowel syndrome and motility disorders. We are making significant strides toward closing the gap on new areas of unmet need. Together with researchers, patient groups and others, we are working to advance scientific research and clinical medicine in GI. About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com . References SOURCE: Takeda Pharmaceutical Co

Clinical ResultPhase 3Drug ApprovalCell Therapy

11 Jul 2024

·www.echemi.com

Takeda's 2023 Fiscal Year: Navigating Challenges and Embracing Transformation

According to Takeda's 2023 fiscal year business report, the company's annual total revenue reached 4,263.8 billion yen, an increase of 5.9% year-over-year. However, net profit declined by 54.6% to 144.1 billion yen. This decline was primarily attributed to the loss of exclusivity for Azilva (a hypertension drug) and Vyvanse (used to treat ADHD and binge eating disorder), as well as the failed development of Alofisel (a stem cell therapy for Crohn's disease) and Exkivity (a lung cancer tyrosine kinase inhibitor). Faced with these challenges, Takeda has announced a corporate restructuring to improve profitability in the coming years. The company expects to incur a one-time restructuring cost of 140 billion yen (around $9 billion) in 2024. Takeda stated that the restructuring measures will involve employee optimization, cost-cutting, and pipeline adjustments, with the goal of increasing the core profit margin by 1% to 2.5% annually, ultimately reaching a level of 30%. As part of the pipeline adjustments, Takeda has discontinued a total of 18 products, including several cancer treatment products such as Exkivity (lung cancer), TAK-981 (cancer therapy), TAK-007 (cell therapy), TAK-573 (multiple myeloma and solid tumors), TAK-102/TAK-103 (solid tumors), and TAK-940 (multiple myeloma). Following the restructuring, Takeda plans to fill the gaps by introducing high-quality products. Orserdulatinib, a third-generation oral BCR-ABL tyrosine kinase inhibitor already approved in China, has been selected as an acquisition target. The drug is used to treat chronic myeloid leukemia with the T315I mutation and patients who are resistant and/or intolerant to first- and second-generation TKIs. According to a report by Asieris Pharmaceuticals, the cumulative sales of orserdulatinib have already exceeded 300 million RMB, and its sales growth has been significant after being covered by medical insurance, indicating its vast market potential. On the other hand, AbbVie's acquisition strategy has been focused on its strengths in the immunology field. Humira, once the world's best-selling drug, has ceded its "drug king" status to Merck's Keytruda (pembrolizumab) due to patent expiration and biosimilar competition. Analysts note that pharmaceutical companies need to adjust their strategies in a timely manner to address the rise of new "drug kings" and the challenges posed by upcoming patent expirations. Currently, the acquisition of biotech companies to supplement product pipelines has become a common strategy among multinational pharmaceutical companies. While ensuring alignment with their own strategic goals, these companies conduct in-depth assessments of biotech companies' pipeline assets and innovation value to optimize their industrial layouts. Analysts emphasize that for large multinational pharmaceutical companies, acquiring "external innovation" through mergers and licensing is just as critical as "internal R&D." Approximately one-third of global innovative drug revenue comes from internal R&D, while two-thirds comes from mergers and collaborations. However, some companies' reliance on external innovation is significantly higher than the average, reaching 60% to 70%. Analysts further note that since 2023, innovative products from China have frequently appeared in transactions involving multinational pharmaceutical companies, indicating that domestic Chinese pharmaceutical companies and their products are gradually gaining international recognition, a positive sign.

AcquisitionCell TherapyImmunotherapyDrug Approval

100 Deals associated with Darvadstrocel ( TiGenix)

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Rectal Fistula	CH	Takeda Pharmaceutical Co., Ltd.	15 Feb 2019
Perianal fistula	EU	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	IS	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	LI	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	NO	Takeda Pharma A/S	23 Mar 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Perianal fistula due to Crohn's disease	Phase 3	AT	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	BE	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	FR	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	DE	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	IL	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	IT	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	NL	Tigenix SAU	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	ES	Tigenix SAU	01 Jul 2012
Crohn Disease	Phase 3	US	Tigenix SAU	-
Colitis, Ulcerative	Phase 2	ES	Hospital Universitario La Paz	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03279081 (ADMIRE-CD II \| ADMIRE-CD-II, CTgov)	Phase 3	Perianal fistula due to Crohn's disease	568	Placebo (Placebo)	pkdhlwebmv(igcoubviwu) = okmahzozrf dyynvbfgrt (jvlmbohxfq, umpqaaurgw - lwjwkbejhm) View more	-	19 Sep 2024
				Cx601 (Cx601)	pkdhlwebmv(igcoubviwu) = ywooxcjyae dyynvbfgrt (jvlmbohxfq, hatkptteaa - xlzaskwide) View more
DDW2024	Not Applicable	Perianal fistula due to Crohn's disease	-	Darvastrocel	rkpvivyssp(chjxpwgsgd) = The Perianal Crohn's Disease Index was significantly lower 3, 6, and 12 months after application in the healed compared to failed group - 1 (0-9), 0 (0-8) and 0 (0-9) vs. 7 (1-13, 8 (0-12) and 6 (0-12), p<0.005 texrvctabu (txzxrvzpoz )	-	21 May 2024
NCT05742100 (PRIME, Pubmed \| Dis Colon Rectum) Manual	Not Applicable	Perianal fistula due to Crohn's disease	73	Darvadstrocel	vlpwedqwbn(vjvupgjsft) = No serious adverse events were reported wadeyeitju (gupqzmxukf )	Positive	11 Apr 2024
NCT03279081 (ADMIRE-CD II, Corporate Publications) Manual	Phase 3	Perianal fistula due to Crohn's disease	-	Alofisel	hjairkgxqa(bzrejojhcn) = did not meet its primary endpoint of combined remission at 24 weeks, based on topline data tnzrevbirz (zexvapbrju ) Not Met	Negative	17 Oct 2023
				Placebo
NCT03706456 (Pubmed) Manual	Phase 3	Crohn Disease \| Perianal fistula	22	Darvadstrocel	hfrdmswghb(deuqxuopkz) = xkkilrxtxc tpshrlzowk (llkytnsdrj, 38.5 - 79.6) View more	Positive	23 Sep 2022
NCT01541579 (ADMIRE-CD, Pubmed \| Dis Colon Rectum)	Phase 3	Perianal fistula due to Crohn's disease	40	Darvadstrocel	frzitfgtzz(djbxmsrevn) = Through Week 104, 7 treatment-emergent serious adverse events were reported, of which 4 occurred between Weeks 52 and 104 zvmwtlddmu (omreyjgbao )	-	07 Dec 2021
				Control (saline solution)
NCT03706456 (CTgov)	Phase 3	Perianal fistula due to Crohn's disease	22	Darvadstrocel	mbxthcsqmq(pttwzbrbyl) = tpfpwhqyof khgqppufxk (yqkxlgjiwr, pegkdhpjuh - swscpufejb) View more	-	07 Oct 2021
UEGW2020	Not Applicable	Perianal fistula	-	Darvadstrocel	jhyyxluyjt(jdkjicfzpl) = One AESI in each group (DVS: benign tumor not product related and control: malignant tumor) was reported during the study period. ffautaevce (bsmkcghcmq ) View more	-	01 Oct 2020
				Darvadstrocel (Control)
NCT01541579 (Pubmed \| Lancet) Manual	Phase 3	Crohn Disease \| Perianal fistula	212	Cx601	uuxbkhkhmg(zcmmaziswp) = zaylreigbb mwygyhpoik (nuecxczrkp ) View more	Positive	24 Sep 2016
				Placebo	uuxbkhkhmg(zcmmaziswp) = ospdblamky mwygyhpoik (nuecxczrkp ) View more

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