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


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  • ||||||||||  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
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Enrollment closed, Viral vector:  A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  May 3, 2020   
    P2,  N=10, Active, not recruiting, 
    NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987 Recruiting --> Active, not recruiting
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Phase classification, Enrollment change, Trial primary completion date, Gene therapy:  TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Dec 20, 2018   
    P1/2,  N=20, Active, not recruiting, 
    NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987 Phase classification: P2 --> P1/2 | N=14 --> 20 | Trial primary completion date: Apr 2023 --> Apr 2018
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Phase classification, Enrollment change, Trial primary completion date, Gene therapy:  TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Mar 24, 2017   
    P2,  N=14, Active, not recruiting, 
    Phase classification: P3 --> P2 Phase classification: P1/2 --> P2 | N=20 --> 14 | Trial primary completion date: Apr 2018 --> Apr 2023
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Enrollment closed, Trial primary completion date, Gene therapy:  TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Apr 21, 2015   
    P1/2,  N=20, Active, not recruiting, 
    Phase classification: P1/2 --> P2 | N=20 --> 14 | Trial primary completion date: Apr 2018 --> Apr 2023 Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Oct 2017 --> Apr 2018
  • ||||||||||  Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
    Enrollment status, Trial primary completion date, Gene therapy:  TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) -  Dec 9, 2014   
    P1/2,  N=20, Enrolling by invitation, 
    Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Oct 2017 --> Apr 2018 Recruiting --> Enrolling by invitation | Trial primary completion date: Dec 2015 --> Oct 2017