Afamitresgene autoleucel Medicine 07 June 2022 Extended indication Treatment of HLA-A*02 positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the MAGE-A4 tumor antigen Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Afamitresgene autoleucel Domain Oncology Reason of inclusion New medicine (specialité) Main indication Oncology other Extended indication Treatment of HLA-A*02 positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the MAGE-A4 tumor antigen Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Adaptimmune Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Dit betreft een genetisch gemodificeerd autoloog melanoma-associated antigen 4 (MAGE A4) specifieke T cell therapie. Complementarity-determining regions (CDR) van de T-cellen van de patiënt worden gemodificeerd waardoor de affiniteit voor de tumor cellen wordt vergroot. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date 2022 Expected Registration 2023 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks 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 In de fase 2, open-label studie (SPEARHEAD-1; NCT04044768), waarin 32 patiënten afamitresgene autoleucel ontvingen werd effectiviteit en veiligheid aangetoond. Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT04044768 Additional remarks Eenmalige infusie van autologe genetisch gemodificeerd afamitresgene autoleucel . Dosis: 1.0 x10^9 tot 10x10^9 cellen 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; https://www.medpagetoday.com/meetingcoverage/ctos/89828 Additional remarks In 2016 waren er 34 wekedelensarcomen van de extremiteiten gediagnosticeerd, waar het synoviaal sarcoom er een van is. De incidentie van dit synoviaal sarcoom wordt geschat op 1 in de 3 miljoen, ongeveer 6 per jaar in Nederland. De schattingen lopen uiteen maar expressie van MAGE-A4 komt voor in ongeveer 80% van de gevallen van synoviaal sarcoom (5 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 No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Gastric cancer; Head and neck cancer; Myxoid liposarcoma; Oesophageal cancer References Adis Insight Additional remarks Other information Additional remarks