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


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  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Atidarsagene Autotemcel, a European Post-Regulatory Approval Model for Delivery of Autologous Hematopoietic Stem Cell Gene Therapy Products Via a Network of Qualified Treatment Centers (QTCs) () -  Dec 16, 2022 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_924;    
    As of 01 July 2022 the European QTCs, listed alphabetically, include: 1) France, Paris, Hospital Robert-Debré with Hospital Saint Louis 2) Germany, Tübingen, Tübingen University Hospital 3) Italy, Milan, Ospedale San Raffaele - TIGET (the center that originally developed arsa-cel and which served as the clinical trials site ) 4) Netherlands, Utrecht Princess Máxima Center with University Medical Center Utrecht and 5) United Kingdom, Manchester, Royal Manchester Children's Hospital. A limited distribution model incorporating qualification of multiple COEs generates a network of QTCs facilitating expanded delivery of autologous HSC-GT to MLD patients.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Trial completion date, Trial primary completion date:  OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Nov 22, 2022   
    P3,  N=6, Recruiting, 
    A limited distribution model incorporating qualification of multiple COEs generates a network of QTCs facilitating expanded delivery of autologous HSC-GT to MLD patients. Trial completion date: Jan 2032 --> Mar 2031 | Trial primary completion date: Jan 2032 --> Mar 2025
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Journal:  The importance of early diagnosis and views on newborn screening in metachromatic leukodystrophy: results of a Caregiver Survey in the UK and Republic of Ireland. (Pubmed Central) -  Nov 12, 2022   
    One parent expressed their gratitude for an early diagnosis as a result of familial MLD screening offered at birth and how it had changed their child's future: "It did and it absolutely has I will be forever grateful for his early diagnosis thanks to his older sister."The rapid rate of deterioration in MLD makes it an essential candidate for NBS, particularly now the first gene therapy (Libmeldy™) has been approved by the European Medicines Agency. Libmeldy™ has also been recommended as a treatment option in England and Wales by the National Institute for Health and Care Excellence (NICE) and is being made available to patients in Scotland via the Scottish Medicines Consortium's ultra-orphan pathway.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Trial completion date, Trial primary completion date, Viral vector:  A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Nov 9, 2022   
    P2,  N=10, Active, not recruiting, 
    Libmeldy™ has also been recommended as a treatment option in England and Wales by the National Institute for Health and Care Excellence (NICE) and is being made available to patients in Scotland via the Scottish Medicines Consortium's ultra-orphan pathway. Trial completion date: Aug 2028 --> Apr 2028 | Trial primary completion date: Aug 2022 --> Apr 2022
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Trial completion date, Gene therapy:  TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Nov 9, 2022   
    P1/2,  N=20, Active, not recruiting, 
    Trial completion date: Aug 2028 --> Apr 2028 | Trial primary completion date: Aug 2022 --> Apr 2022 Trial completion date: Apr 2023 --> Mar 2025
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Rethymic (allogeneic processed thymus tissue-agdc) / Sumitomo Dainippon, Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Global Pricing, Reimbursement and Market Access Trends for Regenerative Medicines, Atmps, Cell & Gene Therapies (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1863;    
    Type of therapy impacts access, with CAR-Ts and gene therapies for rare paediatric conditions more likely to be approved and reimbursed across jurisdictions relative to “tissue engineered therapies.” The value of cell/gene therapies is being recognized (e.g., France has 6 of the 9 evaluated therapies with ASMR II/III and Italy has designated 7 of the 10 as “innovative status”). Visible ex-factory prices of recently introduced cell and gene therapies continue to present new and different challenges to bio/pharmaceutical budgets.
  • ||||||||||  Rethymic (allogeneic processed thymus tissue-agdc) / Sumitomo Dainippon, Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Pricing, Reimbursement and Market Access Trends for Cell and Gene Therapies in the US and Europe (Virtual) -  Mar 8, 2022 - Abstract #ISPOR2022ISPOR_562;    
    There is a huge variation in access with Germany leading the way with largest number of reimbursed therapies (77%) and Spain lagging behind (only 38%). Value of cell/gene therapies is being recognized in France as evidence by 6 of the 9 evaluated therapies being assigned ASMR II/III and 5 of the 8 assessed therapies in Italy being assigned innovative status.
  • ||||||||||  Libmeldy (atidarsagene autotemcel) / Orchard Therap
    The Societal Costs of Metachromatic Leukodystrophy (MLD) in the Netherlands (In-person & Virtual) -  Mar 8, 2022 - Abstract #ISPOR2022ISPOR_334;    
    Prior to the approval of atidarsagene autotemcel (arsa-cel), patients with early-onset MLD had no disease modifying treatment options and very poor survival outcomes, often reaching a decerebrated state before adolescence...In an arsa-cel treated patient, the productivity gains accrued over a working life were estimated at €317,484. There are significant productivity gains for patients treated with arsa-cel compared to untreated patients who do not have opportunities to enter the workforce; significant family income is lost through non-treatment particularly in the later stages of MLD.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Libmeldy (atidarsagene autotemcel) / Orchard Therap
    Review, Journal:  Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord. (Pubmed Central) -  Oct 27, 2021   
    Shortly after EMA approved Libmeldy®, an ex vivo gene therapy with lentivirus vector-transduced autologous CD34-positive stem cells, for treatment of metachromatic leukodystrophy...Based on the large number of planned, ongoing, and completed clinical trials for rare genetic central nervous system diseases, it can be expected that several novel gene therapies will be approved and become available within the near future. Essential for this to happen is the in depth characterization of short- and long-term effects, safety aspects, and pharmacodynamics of the applied gene therapy platforms.
  • ||||||||||  NICE, Reimbursement, Review, Journal:  Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review. (Pubmed Central) -  Aug 21, 2021   
    There were ten gene therapy products (talimogene laherparepvec [TA410], strimvelis [HST7], tisagenlecleucel [TA554 and TA567], axicabtagene ciloleucel [TA559], voretigene neparvovec [HST11], autologous anti-CD19-transduced CD3+ cells [TA677], betibeglogene autotemcel [ID968], onasemnogene abeparvovec [HST15] and OTL-200 [ID1666]), one tissue engineered product (holoclar [TA467]) and three somatic cell therapy products (darvadstrocel [TA556] and autologous chondrocyte implantation [ACI] [TA477 and TA508])...In conclusion, the challenges raised by the economic appraisal of ATMPs, albeit not unique, may be exacerbated by the uncertainty related to the often scant evidence base. Adaptations of the conventional decision-making process rather than completely new methods may improve appraisals of ATMPs.
  • ||||||||||  OTL-200 / Orchard Therap
    [VIRTUAL] The Cost-Effectiveness of OTL-200 for the Treatment of Metachromatic Leukodystrophy (MLD) () -  Apr 12, 2021 - Abstract #ISPOR2021ISPOR_1165;    
    Subgroup analyses indicated OTL-200 was more cost-effective for patients treated pre-symptomatically. CONCLUSIONS : This is the first de novo cost-effectiveness study which has considered both the motor and cognitive aspects of MLD and generated ICERs which are substantially lower than the cost-effectiveness thresholds used in a number of jurisdictions, indicating that OTL-200 is a cost-effective use of resources.
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Trial initiation date:  OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Oct 5, 2020   
    P3,  N=6, Recruiting, 
    CONCLUSIONS : This is the first de novo cost-effectiveness study which has considered both the motor and cognitive aspects of MLD and generated ICERs which are substantially lower than the cost-effectiveness thresholds used in a number of jurisdictions, indicating that OTL-200 is a cost-effective use of resources. Initiation date: Sep 2020 --> Dec 2020