Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin 
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 7 Diseases   4 Trials   4 Trials   44 News 


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  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    UK Experience of Managing Metachromatic Leukodystrophy in the Advent of Stem Cell Gene Therapy (Poster 1 Annex Hall) -  Aug 28, 2025 - Abstract #ICIEM2025ICIEM_1448;    
    Atidarsagene autotemcel, an autologous haematopoietic stem cell gene therapy, is approved in the UK for the management of pre-symptomatic LI and pre-symptomatic or early symptomatic early juvenile (EJ) MLD...Pre-symptomatic cases can be identified through screening family members. Ultimately, establishing newborn screening must be a priority.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    UK Experience of Managing Metachromatic Leukodystrophy in the Advent of Stem Cell Gene Therapy (Poster 1 Annex Hall) -  Aug 28, 2025 - Abstract #ICIEM2025ICIEM_780;    
    Atidarsagene autotemcel, an autologous haematopoietic stem cell gene therapy, is approved in the UK for the management of pre-symptomatic LI and pre-symptomatic or early symptomatic early juvenile (EJ) MLD...Pre-symptomatic cases can be identified through screening family members. Ultimately, establishing newborn screening must be a priority.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Myeloid-mediated enzymatic correction of ARSA-deficient neural cells in hematopoietic stem cell gene therapy for metachromatic leukodystrophy (Fibes 2) -  Aug 21, 2025 - Abstract #ESGCT2025ESGCT_1242;    
    Following over ten years of safety and efficacy data, HSC-GT (arsa-cel) received marketing authorization in Europe and the UK (Libmeldy), and recently in the USA (Lenmeldy) for the treatment of pre-symptomatic late infantile (LI), pre-symptomatic early juvenile (EJ), or early symptomatic EJ MLD patients...The transgenic ARSA enzyme undergoes phosphorylation of mannose-6 (M6P) residues, and the M6P receptor partly mediates its uptake by MLD neural cells. Our findings underscore effective myeloid-mediated enzymatic cross-correction of ARSA-deficient neurons and glial cells within a clinically relevant HSC GT framework, offering insights into the mechanisms of this GT approach and supporting its broader therapeutic potential for other LSDs.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Biological properties and clonality of engineered hematopoietic stem/progenitor cells persisting long-term after gene therapy (Fibes 2) -  Aug 21, 2025 - Abstract #ESGCT2025ESGCT_1072;    
    Our findings underscore effective myeloid-mediated enzymatic cross-correction of ARSA-deficient neurons and glial cells within a clinically relevant HSC GT framework, offering insights into the mechanisms of this GT approach and supporting its broader therapeutic potential for other LSDs. Thus, we are studying the functional features, clonality and maintenance of stemness properties of LV-transduced HSPC in long-term GT (LTGT) patients with Wiskott-Aldrich Syndrome (WAS, n=6 treated with etuvetidigene autotemcel) and Metachromatic Leukodystrophy (MLD, n=10 treated with atidarsagene autotemcel) reaching a follow-up of >8 years post-GT (follow-up range: 2
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Review, Journal:  Treatment of leukodystrophies: Advances and challenges. (Pubmed Central) -  May 30, 2025   
    Gene therapy has become a viable option, with ex vivo approaches like atidarsagene autotemcel providing promising outcomes for early-onset MLD...Despite these advances, challenges remain, including the ultrarare nature of most leukodystrophies, limited natural history data, high treatment costs, and barriers to accessibility. Future developments, including newborn screening and close international collaboration, aim to enhance early diagnosis, refine treatment timing, and expand access to innovative therapies.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    BIOLOGICAL PROPERTIES AND CLONALITY OF ENGINEERED HEMATOPOIETIC STEM/PROGENITOR CELLS PERSISTING LONG-TERM AFTER GENE THERAPY (Poster Hall) -  May 15, 2025 - Abstract #EHA2025EHA_1782;    
    Moreover, we provided evidence that the engrafted pool of engineered HSPC retain self-renewal properties preserving their number and clonality up to 10 years post-GT. Finally, scRNAseq data suggest that primitive HSC from LT-GT patients express molecular signature associated with stemness.Overall, this study will shed light on human engineered HSPC to maintain a functional and safe long-term graft.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD) (New Orleans Theater C) -  Apr 10, 2025 - Abstract #ASGCT2025ASGCT_1830;    
    P1/2, P2
    Given the stark difference in motor outcomes between the treated and untreated sibling pairs, this analysis reinforces the importance of early diagnosis through newborn screening to enable all early-onset patients to have the opportunity for pre-symptomatic treatment, not just the younger siblings of symptomatic patients. Clinical trial registration: NCT01560182, NCT03392987, EudraCT 2009-017349-77, EudraCT 2017-001730-26 Disease Focus of Abstract:Storage/Lysosomal Disorders
  • ||||||||||  Imlygic (talimogene laherparepvec) / Amgen, Provenge (sipuleucel-T) / Bausch Health, Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Diversity, Equity, and Inclusion in Gene and Cell Therapies Clinical Trials () -  Mar 24, 2025 - Abstract #ISPOR2025ISPOR_431;    
    The findings revealed persistent racial disparities in gene and cell therapy clinical trials, with minority populations underrepresented and White participants comprising the majority. Achieving DEI in clinical trials remains a challenge, underscoring the need for greater efforts to address these disparities.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Trial completion date:  A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Jan 28, 2025   
    P2,  N=10, Active, not recruiting, 
    Given the stark difference in motor outcomes between the treated and untreated sibling pairs, this analysis reinforces the importance of early diagnosis through newborn screening to enable all early-onset patients to have the opportunity for pre-symptomatic treatment, not just the younger siblings of symptomatic patients. Trial completion date: Apr 2028 --> Jan 2026
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    FDA Comments - Lenmeldy (HCC Ballroom C; In-Person) -  Dec 12, 2024 - Abstract #TCTASTCTCIBMTR2025TCT_ASTCT_CIBMTR_638;    
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Lenmeldy (HCC Ballroom C; In-Person) -  Dec 12, 2024 - Abstract #TCTASTCTCIBMTR2025TCT_ASTCT_CIBMTR_637;    
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    From Pipeline to Pricing & Reimbursement: Economic Impact and Access Challenges of Upcoming Gene Therapies () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2968;    
    Rare indications still dominate the pipeline, with an ever increasing price- Lenmeldy recently launched at $4.25m in the US- posing ongoing affordability and access challenges. However, the emergence of gene therapies targeting indications with larger prevalences, such as diabetes and Parkinson
  • ||||||||||  Finding a Way for Patients to Access Gene Therapies () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2243;    
    Canada and UK mainly use financial-based schemes built on simple price discounts. Australia and the US aim at implementing schemes whereby treatment cost payers incur corresponds to expected health outcome for a particular patient.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Unlocking Equitable Access to Revolutionary Cell and Gene Therapies Across EU Member States () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_304;    
    This is likely due to smaller target populations and the high price of C>s reducing affordability and pushing the incremental cost-effectiveness ratios above accepted thresholds. Although economic discussions have been shown to be a tool for resource rationalization and efficiency, their impact on equity may need to be re-considered.
  • ||||||||||  Drivers of Access to Gene Therapies in Poland and Selected Central and Eastern Europe Countries () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_93;    
    The availability of GTx in selected CEE countries is limited. Considering there are 88 ongoing GTx phase 3 clinical trials, with 58 expected to be completed within the next 5 years, the decision-makers will likely face even greater challenges to balance patient access and impact on public budgets.
  • ||||||||||  Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin, Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Journal:  Marketing authorisation for rare diseases: The European regulatory perspective using the example of gene and cell therapies (Pubmed Central) -  Oct 2, 2024   
    The role of clinical data obtained under everyday conditions (real world data) to support the generation of evidence in the pre- and post authorisation phase is critically examined. Furthermore, the paper outlines aspects related to conditional versus standard marketing authorisations as well as aspects related to registry-based non-interventional studies in the context of market and patient access to urgently needed drugs.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    LENTIVIRAL HEMATOPOIETIC STEM CELL GENE THERAPY FOR LATE JUVENILE METACHROMATIC LEUKODYSTROPHY (Poster Room | Level 0) -  Jul 30, 2024 - Abstract #SSIEM2024SSIEM_430;    
    P3
    Atidarsagene autotemcel ("arsa-cel") is a hematopoietic stem cells gene therapy (HSC-GT) consisting of autologous CD34+ cells transduced ex vivo with a lentiviral vector encoding for the human ARSA gene, infused after intravenous myeloablative busulfan conditioning. This analysis shows that the safety profile and pharmacodynamic efficacy of arsa-cel in LJ-MLD were similar to those observed in previous trials in early-onset MLD.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    NEWBORN SCREENING (NBS) FOR METACHROMATIC LEUKODYSTROPHY - A PROSPECTIVE STUDY (Camellia Auditorium | Level 0) -  Jun 13, 2024 - Abstract #SSIEM2024SSIEM_70;    
    A three-tiered screening approach demonstrated its technical feasibility. This pilot study effectively detected four newborns, allowing for a timely administration of treatment.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Journal:  Access in all areas? a round up of developments in market access and health technology assessment: part 4. (Pubmed Central) -  Apr 22, 2024   
    In conclusion, this pre-pilot study adds to the international evidence that recommends newborn screening for MLD, making it possible for patients to benefit fully from treatment through early diagnosis. In this latest update, we look at recent developments in market access including the pricing agreement of Libmeldy
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Kyowa Kirin
    LENTIVIRAL HAEMATOPOIETIC STEM CELL GENE THERAPY (ATIDARSAGENE AUTOTEMCEL) FOR LATE JUVENILE METACHROMATIC LEUKODYSTROPHY (MLD) (BOISDALE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1916;    
    P3
    Atidarsagene autotemcel (arsa-cel) is a haematopoietic stem cell (HSC) gene therapy (GT) and consists of autologous CD34+ cells transduced ex vivo with a lentiviral vector encoding for the human ARSA gene, infused after intravenous myeloablative busulfan conditioning...Stem cell source was mobilized peripheral blood collected after G-CSF (6-8 doses) and Plerixafor (administered 6-8 hours before the leukapheresis)... This analysis demonstrates that the initial results on engraftment, ARSA activity, safety, and drug product characteristics for arsa-cel in LJ-MLD were similar to those reported in early-onset (late infantile and early juvenile) MLD
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Kyowa Kirin
    ATIDARSAGENE AUTOTEMCEL (HAEMATOPOIETIC STEM CELL GENE THERAPY) PRESERVES COGNITIVE AND MOTOR DEVELOPMENT IN METACHROMATIC LEUKODYSTROPHY WITH UP TO 12 YEARS FOLLOW-UP (CLYDE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_991;    
    P1/2, P2
    Clinical Trial Registry: NCT01560182, NCT03392987, EudraCT 2009-017349-77 Background: Metachromatic leukodystrophy (MLD) is a rare neurometabolic disorder caused by deficiency of arylsulfatase A (ARSA) enzyme, leading to accumulation of toxic sulfatides in the central and peripheral nervous systems, and subsequent progressive demyelination, neurodegeneration, loss of motor and cognitive function, and early death. With up to 12 years follow-up in the earliest treated patient and a cumulative 251 patient-years of follow-up, arsa-cel shows a favorable benefit-risk profile with clinically meaningful and sustained efficacy, preventing severe motor and cognitive impairment, preserving speech, and slowing disease progression in early-onset MLD patients
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Journal:  Atidarsagene autotemcel for metachromatic leukodystrophy. (Pubmed Central) -  Feb 3, 2024   
    With up to 12 years follow-up in the earliest treated patient and a cumulative 251 patient-years of follow-up, arsa-cel shows a favorable benefit-risk profile with clinically meaningful and sustained efficacy, preventing severe motor and cognitive impairment, preserving speech, and slowing disease progression in early-onset MLD patients No abstract available
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Atidarsagene Autotemcel (Hematopoietic Stem Cell (MTCC - Exhibit Halls DE) -  Jan 10, 2024 - Abstract #ACMG2024ACMG_754;    
    After busulfan conditioning, arsa-cel was administered intravenously. With up to 12 years follow-up in the earliest treated patient and a cumulative 251 patient-years of follow-up, arsa-cel shows a favorable benefit-risk profile with clinically meaningful, sustained efficacy, preventing severe motor and cognitive impairment, preserving speech, and slowing disease progression in early-onset MLD patients.
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Retrospective data, Journal:  A retrospective cohort study of Libmeldy (atidarsagene autotemcel) for MLD: What we have accomplished and what opportunities lie ahead. (Pubmed Central) -  Sep 13, 2023   
    The success of HSC-GT for MLD has heralded a new era of hope for families affected by this devastating disease, yet currently, most patients are ineligible for treatment at diagnosis. The feasibility of apheresis in infants and the availability of a licenced, effective HSC-GT product highlights the urgent need for newborn screening to ensure that patients can be diagnosed and treated before symptom onset.