- |||||||||| 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;
- |||||||||| 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.
- |||||||||| 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.
- |||||||||| 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
Treatment of metachromatic leukodystrophy with atidarsagene autotemcel - autologous lentiviral hematopoietic stem-cell gene therapy - in the EU: A case report (Foyer 3B, 3rd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_773; After confirmation of eligibility, treatment was initiated at the QTC at 9 months of age, and arsa-cel was administered to the child at 11 months of age. After haematological recovery (neutrophil and platelet engraftment on day +31 and + 28 post-GT, respectively), the child is continuing follow up at the QTC as an out-patient with clinical and laboratory examinations twice a week until approximately 90 days post-GT.This is the first case of an MLD patient treated in Europe with arsa-cel according to the European cross-border healthcare legislation, and made possible by the support of an external team facilitating the approval process and its implementation, especially in communication with the Company, the local center, the QTC, and the state health authorities.
- |||||||||| Libmeldy (atidarsagene autotemcel) / Orchard Therap
The Desirability of Implementing an Outcome-Based Delayed Payment Model for Autologous Gene Therapy Atidarsagene Autotemcel (LIBMELDY () - Mar 9, 2023 - Abstract #ISPOR2023ISPOR_382; Outcome-based models were suitable to mitigate the financial risk and are preferred, from the payer perspective, for AA over simple discounts in situations when clinical performance was similar to or worse than predicted. The framework and calculation tool can aid reimbursement decision-makers to weigh the desirability of each of the different payment scenarios and support them with negotiating and implementing an outcome-based spread payment model.
- |||||||||| Libmeldy (atidarsagene autotemcel) / Orchard Therap
Journal: Atidarsagene autotemcel for metachromatic leukodystrophy. (Pubmed Central) - Feb 27, 2023 This new therapeutic consists of patients' CD34+ hematopoietic stem/progenitor cells (HSPCs) transduced with a lentiviral vector encoding functional ARSA cDNA. The gene-corrected cells get reinfused into the patients after a cycle of chemotherapy conditioning.
- |||||||||| Libmeldy (atidarsagene autotemcel) / Orchard Therap
WHAT WE HAVE DONE AND WHAT WE HAVE FAILED TO DO: THE STORY OF LIBMELDY AS A NOW LICENSED STEM CELL GENE THERAPY FOR MLD (MAILLOT) - Feb 11, 2023 - Abstract #EBMT2023EBMT_849; Our real-world experience highlights that most patients with MLD present with disease features and are ineligible for treatment at diagnosis. The feasibility of apheresis in very young and low weight infants together with the availability of a licensed and effective HCT-GT product indicate the urgent need for newborn screening for MLD to ensure that patients can be diagnosed and treated before the onset of symptomatic clinical disease.
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