Tabelecleucel Medicine 12 June 2019 Extended indication Ebvallo is indicated as monotherapy for treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. Therapeutic value No estimate possible yet Total cost Registration phase Positive CHMP opinion Product Active substance Tabelecleucel Domain Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Ebvallo is indicated as monotherapy for treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. Current proprietary name Already available biosimilars / generics Proprietary name Ebvallo Manufacturer Atara Portfolio holder Pierre Fabre Mechanism of action Allogeneic modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date October 2021 Expected Registration December 2022 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen oktober 2022. Therapeutic value Current treatment options Rituximab Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De huidige behandeling is redelijk effectief. Er zijn nog geen resultaten uit de studie bekend. Mogelijk is er weinig vraag naar een nieuwe behandeloptie. Duration of treatment Average 3 month / months Frequency of administration 3 times every 5 weeks Dosage per administration References NCT03394365 (ALLELE) Additional remarks Tabelecleucel wordt toegediend in cycli van 5 weken (35 dagen). Gedurende elke cyclus zal tabelecleucel worden toegediend in een dosis van 2×10^6 cellen/kg op dag 1, 8 en 15. Indien nodig worden meerdere behandelcycli gevolgd. De cut-off data in de ALLELE studie kwam uit op gemiddeld 7,56 doseringen. Expected patient volume per year Patient volume 23 - 35 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References (1) Declaratiegegevens; 2) Garcia-Cadenas et al. 2019; Eur J Haematol; 3) Jagadeesh et al. 2020 J Clin Oncol; 4) Trappe et al. 2017, J Clin Oncol Additional remarks Op basis van declaratiegegevens uit 2020 voor rituximab bij de indicatie: Behandeling van post-transplantatie lymfoproliferatieve ziekte (PTLD) na orgaan- of stamcel transplantatie bij volwassenen, is de inschatting dat er maximaal 71 mensen rituximab krijgen tegen PTLD (1). Het is niet bekend welk type transplantatie deze patiënten heeft gehad. Dat is relevant aangezien patiënten met stamceltransplantaties (HCT) vaker rituximab refractair zijn (50%) dan orgaantransplantatie (SOT) patiënten (33%) (2,3,4). Dit bevindt zich dus ergens tussen 23 en 35 patiënten. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References www.clinicaltrials.gov. Additional remarks Other information Additional remarks