Letetresgene autoleucel Medicine 08 June 2021 Extended indication Letetresgene autoleucel in combination with fludarabine and cyclophosphamide for second line or later treatment of advanced (metastatic or unresectable) HLA-A2-positive, NY-ESO1-positive and/or LAGE-1a-positive myxoid/round cell liposarcoma or Synovial sarcoma in children over 10 years of age, adolescents, adults and elderly who have disease progression following treatment with anthracycline based chemotherapy. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Letetresgene autoleucel Domain Oncology Reason of inclusion New medicine (specialité) Main indication Oncology other Extended indication Letetresgene autoleucel in combination with fludarabine and cyclophosphamide for second line or later treatment of advanced (metastatic or unresectable) HLA-A2-positive, NY-ESO1-positive and/or LAGE-1a-positive myxoid/round cell liposarcoma or Synovial sarcoma in children over 10 years of age, adolescents, adults and elderly who have disease progression following treatment with anthracycline based chemotherapy. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Adaptimmune Portfolio holder Mechanism of action Autologous modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks NY-ESO-1 autologous engineered TCR-T cells (engineered TCR). Fabrikant: adaptimmuno Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date June 2026 Expected Registration July 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Bestraling en chemotherapie. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Volgens experts een interessant product. Er is nog niet veel te zeggen aan de hand van de resultaten op dit moment. Duration of treatment Frequency of administration Dosage per administration 1 tot 6 x10^9 cellen References NCT03967223 (IGNYTE-ESO) Additional remarks Expected patient volume per year Patient volume 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; https://mens-en-gezondheid.infonu.nl/aandoeningen/187822-synoviaal-sarcoom-kanker-van-weefsel-rond-gewrichten.html#behandeling; Lai et al. Oncoimmunology. 2012 Nov 1; 1(8): 1409–1410. Additional remarks In 2016 waren er 34 wekedelen sarcomen van de extremiteiten gediagnosticeerd, waar het synoviaal carcinoom er een van is. De incidentie van dit synoviaal carcinoom wordt geschat op 1 in de 3 miljoen, ongeveer zes per jaar in Nederland. Expressie van NY-ESO-1 komt voor in ongeveer 80% van de gevallen van synoviaal sarcoom (vijf patiënten). Expected cost per patient per year Cost References Additional remarks Gezien de PRIME-status zal deze behandeling waarschijnlijk hoge kosten met zich meebrengen. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Gastrointestinal cancer, Malignant melanoma, Multiple myeloma, Non-small cell lung cancer, Ovarian cancer, Liposarcoma. References AdisInsight Additional remarks Other information Additional remarks