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Last update 16 May 2025

Darvadstrocel(TiGenix)

Last update 16 May 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Mesenchymal stem cell therapy

Synonyms

Darvadstrocel, CX601, Alofisel

+ [1]

Target-

Action-

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

The Spanish Association of Biotech companies (ASEBIO)

Takeda Pharmaceutical Co., Ltd.

TiGenix NV

+ [1]

Inactive Organization

Hospital Universitario La Paz

Takeda Canada, Inc.

License Organization

Takeda Pharmaceutical Co., Ltd.

TiGenix NV

Mesoblast Ltd.

Drug Highest PhaseApproved

First Approval Date

European Union (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

/ Active, not 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 Apr 2025·United European Gastroenterology Journal

Darvadstrocel for complex perianal fistulas in Crohn's disease: A systematic review and meta‐analysis

Review

Author: López‐García, Olga N. ; García‐Brenes, Miguel A. ; Taxonera, Carlos ; Zapater, Raúl ; Alba, Cristina ; Olivares, David

Abstract:

Background:

Local injection of darvadstrocel, a suspension of expanded adipose‐derived allogenic mesenchymal stem cells, has been used for treatment‐refractory perianal fistulas in Crohn's disease (CD).

Objective:

This study aimed to investigate efficacy and safety of darvadstrocel for complex perianal fistulas in CD.

Methods:

A systematic search was conducted through April 2024 in relevant databases for observational studies evaluating darvadstrocel. A random‐effects meta‐analysis model was used to calculate the pooled effect sizes (proportions or incidence rates [IRs]) with 95% confidence intervals (CIs) of effectiveness and safety outcomes. The risk of bias was evaluated using the Joanna Briggs Institute Critical Appraisal Tool. The I2 value assessed heterogeneity. Sensitivity and subgroup analyses were also conducted.

Results:

Twelve studies were included with 595 patients. The pooled rate of patients achieving clinical remission, defined as fistula healing, was 68.1% at month 6 (95% CI 63.4–72.7) and 77.2% (95% CI 70.1–83.8) at month 12. Combined remission, defined as clinical remission and absence of collections >2 cm confirmed by magnetic resonance imaging, was reported in 60.6% and in 69.7% of patients at months 6 and 12, respectively. The rate of patients with treatment failure, defined as no clinical remission at the last follow‐up (mean 18.7 months; SD 9.9), was 34.5%. Failure rate was independent of follow‐up time (p = 0.85). For effectiveness outcomes, between‐study heterogeneity was negligible. Subgroup analysis indicated that none of the covariates modified the treatment effect. Pooled IRs per 100 patient‐years of adverse events (AE), serious AEs, perianal abscesses, and reoperations were 19.6, 3.2, 16.9 and 7.1, respectively.

Conclusion:

Evidence from observational studies supports the efficacy and safety of darvadstrocel for complex perianal fistulas in CD. Studies have reported high fistula healing rates that can be sustained long‐term in most patients, with negligible between‐study heterogeneity, as well as a favorable safety profile.

05 Mar 2025·Journal of Crohns & Colitis

Corrigendum: Darvadstrocel for Complex Perianal Fistulas in Japanese Adults with Crohn’s Disease: A Phase 3 Study

03 Mar 2025·INFLAMMATORY BOWEL DISEASES

Allogenic Stem Cells in Anal Fistulas of Crohn’s Disease: From Promising Premises to Real Life Experience

Article

Author: Sabbagh, Charles ; Etienney, Isabelle ; Brochard, Charlène ; Siproudhis, Laurent ; Cotte, Eddy ; Bouchard, Dominique ; Zerbib, Philippe ; Fathallah, Nadia

Abstract:

Background:

Allogenic adipocyte stem cells represent an unprecedented opportunity for regenerative therapy to treat Crohn anal fistulas. Apart from the results of one 8-year-old trial, scientific evidence remains scarce.

Methods:

Data from consecutive patients treated with darvadstrocel for Crohn anal fistulas were reviewed at 6 first tertiary reference centers. The judgment criteria combined asymptomatic status plus clinical occlusion of the fistula tract and MRI-confirmed healing of the tract (no inflammation and/or disappearance of the tract). Both clinical and MRI-confirmed healing of the tract defined a deep remission. Clinical remission was defined by an absence of complaint, occlusion of all external openings, and no fistula discharge.

Results:

A total of 116 patients were extracted (median follow-up after cell stem injection: 11 [6-14] months). No severe adverse events were reported after surgery except for subsequent anal surgery in 29 (25%) patients. Fifty-one (44%) patients had clinical remission defined by the absence of complaints, the occlusion of all external openings, and the presence of no fistula discharge. Deep remission was observed in 23 (29%) patients. Patients with clinical remission more often received combined therapy (immunosuppressant antitumor necrosis factors) than those with no improvement (31 of 51 [61%] vs 23 of 65 [35%]; P = .007). Regression analysis showed that high fistulas (odds ratio, 3.8 [1.1-12.5]; P = .03) and younger age (<38 years, odds ratio, 2.3 [1.0-58;4]; P = .02) were associated with a better outcome.

Conclusions:

Allogeneic stem cell treatment of Crohn’s anal fistulas results in complete remission in less than half of patients, with a significant reintervention rate.

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

13 Dec 2024

·www.fiercepharma.com

EMA backs Merck's Welireg, Bayer's ATTR med and Gilead's liver drug from $4.3B deal

Drug reviewers at the European Medicines Agency figured that Eli Lilly doesn't need a separate indication for Mounjaro’s use in sleep apnea. In a flurry of decisions Friday, the European Medicines Agency’s human products committee recommended 17 new drugs for approval while letting Takeda withdraw from market an inflammatory bowel disease complication medication.Merck & Co.’s kidney cancer drug Welireg, Bayer and BridgeBio’s transthyretin amyloid cardiomyopathy (ATTR-CM) therapy acoramidis, and Gilead’s primary biliary cholangitis (PBC) treatment seladelpar were among the 17 for which the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions.For Welireg in third-line clear cell renal cell carcinoma and in tumors associated with von Hippel-Lindau disease, the CHMP is recommending a conditional marketing authorization. Both indications have counterpart approvals from the U.S. FDA.The CHMP based its positive opinion on tumor shrinkage data from the phase 2 LITESPARK-004 trial and the phase 3 LITESPARK-005 study, as well as Welireg’s progression-free survival win against Novartis’ Afinitor in the latter study. However, a final analysis of the -005 trial didn’t spot a statistically significant overall survival benefit for the Merck drug in clear cell kidney cancer despite a favorable trend.Analysts have previously figured that Welireg, which Merck got from a 2019 acquisition, holds blockbuster potential if it can reach earlier kidney cancer settings.Moving to acoramidis, which will bear the brand name Beyonttra once the European Commission adopts a formal marketing authorization. Bayer obtained European rights to the ATTR drug earlier this year by paying BridgeBio $310 million in upfront and near-term milestones. Both stabilizers of transthyretin, acoramidis is a direct competitor to Pfizer’s blockbuster tafamidis franchise. The CHMP endorsement comes after the FDA approved the drug under the commercial moniker Attruby late November. The drug’s U.S. label cites its benefit on the composite measurement of all-cause mortality and cardiovascular-related hospitalizations but not death reduction by itself.For its part, the CHMP in its summary also cited Beyonttra’s improvement on the composite endpoint. Detailed recommendations will be made available following the European Commission’s final decision, which Bayer said is expected in the coming months. Pending approval, Bayer said it plans to launch Beyonttra in Europe in the first half of 2025.As to Gilead’s seladelpar, which gained the FDA’s go-ahead for the treatment of PBC in August, the CHMP reviewed the PPARδ agonist under the agency’s Priority Medicines pathway, which provides extra support for promising medicines with a potential to address unmet medical needs. Seladelpar came to Gilead via the Big Biotech’s $4.3 billion buyout of CymaBay Therapeutics earlier this year.The CHMP’s endorsement of seladelpar, known as Livdelzi in the U.S., comes shortly after a court of the European Union backed the European Commission’s decision to revoke the PBC nod previously granted to Intercept and Advanz Pharma’s Ocaliva.Speaking of market withdrawal, Takeda has decided to pull its Alofisel from the EU market as a treatment for complex anal fistulas in Crohn’s disease. The move comes more than a year after the stem cell therapy was found to be no better than placebo in a phase 3 trial dubbed ADMIRE-CD II. Additional results presented this February again showed no statistical differences in any of the secondary endpoints. Takeda agreed to the study because the original trial supporting the approval, ADMIRE-CD, was relatively small in size with modest benefits observed.According to the EMA, Takeda is voluntarily pulling Alofisel as the company considered it impossible to provide additional data to meet the agency’s requirements.Takeda had months of discussions with the EMA, healthcare professionals and other stakeholders on the totality of data around Alofisel, Ramona Sequeira, head of Takeda’s global portfolio division, said in a statement Friday.“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,” Sequeira said.Takeda said it started reaching out to doctors and others about this development earlier today, and that it’s talking to other health authorities in countries where Alofisel is approved. About 1 in 3 Crohn’s patients would develop a fistula, which is marked by abnormal passages between the lower parts of the gut and the skin near the anus.Alongside those decisions, the CHMP also considered Eli Lilly’s application to expand the label of Mounjaro to cover obstructive sleep apnea in obesity patients. But the drug reviewers figured that Mounjaro’s existing indication for weight management already covers this use and that it does not need a separate indication. Among drugs that earned label expansions from the CHMP, Lilly’s Omvoh, a direct competitor to AbbVie’s blockbuster Skyrizi, won a recommendation for use in Crohn’s disease. Boehringer Ingelheim’s Ofev has become the first med recommended for treating progressive fibrosing interstitial lung disease in children from the age of 6. The condition is rare, affecting less than 5 children per million in the EU. The drug will come in a lower strength for children than for adults.GSK’s PD-1 inhibitor Jemperli, used in combination with chemotherapy, won an extension to treat all first-line patients with primary advanced or recurrent endometrial cancer. The indication was previously limited to mismatch repair deficient or microsatellite instability-high tumors.

Clinical ResultDrug ApprovalPhase 3AcquisitionPhase 2

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	Switzerland	Takeda Pharmaceutical Co., Ltd.	15 Feb 2019
Perianal fistula	European Union	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	Iceland	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	Liechtenstein	Takeda Pharma A/S	23 Mar 2018
Perianal fistula	Norway	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	Austria	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Belgium	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	France	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Germany	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Israel	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Italy	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Netherlands	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Perianal fistula due to Crohn's disease	Phase 3	Spain	The Spanish Association of Biotech companies (ASEBIO)	01 Jul 2012
Crohn Disease	Phase 3	United States	The Spanish Association of Biotech companies (ASEBIO)	-
Colitis, Ulcerative	Phase 2	Spain	Hospital Universitario La Paz	-

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04075825 (CTgov)	Phase 3	Perianal fistula due to Crohn's disease	150	Placebo (Placebo)	traanxcpaz = ujphqdvgwl hsaqhmcnce (vhvlkcvhqm, uezxlaynop - oeqrsikusw) View more	-	23 Apr 2025
				Darvadstrocel (Darvadstrocel)	traanxcpaz = cmbhelckxl hsaqhmcnce (vhvlkcvhqm, morlndhjtm - byuhtvloox) View more
NCT03279081 (ADMIRE-CD II \| ADMIRE-CD-II, CTgov)	Phase 3	Perianal fistula due to Crohn's disease	568	Placebo (Placebo)	aopqrhmpoa = tplpaaqwru zwrrucmauj (shqiskpevd, xfehrozrau - dhtmctaewe) View more	-	19 Sep 2024
				Cx601 (Cx601)	aopqrhmpoa = lpuokllyoj zwrrucmauj (shqiskpevd, hrlrhtdwbe - bvaadngmee) View more
DDW2024	Not Applicable	Perianal fistula due to Crohn's disease	-	Darvastrocel	fhqqrzqfic(oghzjurucd) = 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 ufbrxxangn (dlbqeidxxa )	-	21 May 2024
NCT05742100 (PRIME, Pubmed \| Dis Colon Rectum) Manual	Not Applicable	Perianal fistula due to Crohn's disease	73	Darvadstrocel	ocjgfskpco(imsqnkafnw) = No serious adverse events were reported plqyiptizh (febwvrwfrt )	Positive	11 Apr 2024
NCT03279081 (ADMIRE-CD II, Corporate Publications) Manual	Phase 3	Perianal fistula due to Crohn's disease	-	Alofisel	qtqpvodnha(jhhziamsgn) = did not meet its primary endpoint of combined remission at 24 weeks, based on topline data wohkheozzy (dwbhafwsei ) Not Met	Negative	17 Oct 2023
				Placebo
NCT03706456 (Pubmed) Manual	Phase 3	Crohn Disease \| Perianal fistula	22	Darvadstrocel	eglrinafav(vvcrqrszar) = kbkubsxpwg dslytbmfvb (rvewfvucud, 38.5 - 79.6) View more	Positive	23 Sep 2022
NCT01541579 (ADMIRE-CD, Pubmed \| Dis Colon Rectum)	Phase 3	Crohn Disease	40	Darvadstrocel	yxdrbeiwxj(czizccifdm) = Through Week 104, 7 treatment-emergent serious adverse events were reported, of which 4 occurred between Weeks 52 and 104 zowmbmpoah (clkuzaczux )	-	07 Dec 2021
				Control (saline solution)
NCT03706456 (CTgov)	Phase 3	Perianal fistula due to Crohn's disease	22	Darvadstrocel	fseivthlrn = apestvcztl poanpbqtbw (gzzqjoaawy, irpabngtma - ptppnpcecz) View more	-	07 Oct 2021
UEGW2020	Not Applicable	Crohn Disease \| Fistula	-	Darvadstrocel	gaxpdjguda(emdgxaprpl) = One AESI in each group (DVS: benign tumor not product related and control: malignant tumor) was reported during the study period. fzhrwkhmmu (fexnkowrap ) View more	-	01 Oct 2020
				Darvadstrocel (Control)
NCT01541579 (Pubmed \| Lancet) Manual	Phase 3	Crohn Disease \| Perianal fistula	212	Cx601	lrcdobkivy(kqevcqeexf) = sneauuzlwn ulpjaplpqd (amnftaqxge ) View more	Positive	24 Sep 2016
				Placebo	lrcdobkivy(kqevcqeexf) = guacxmkskl ulpjaplpqd (amnftaqxge ) View more

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