Vocimagene amiretrorepvec Medicine 12 June 2019 Extended indication Recurrent High Grade Glioma Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Vocimagene amiretrorepvec Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Brain cancer Extended indication Recurrent High Grade Glioma Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Tocagen Portfolio holder Mechanism of action Gene therapy Route of administration Intracerebral Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Combinatie van een eenmalige behandeling door injectie in het geresecteerde weefsel en een tablet. Vocimagene amiretrorepvec is een gentherapie wat het gen voor cytosine deaminase (CD) in cellen incorporeert via een replicerend, non-lytisch retrovirale vector. Deze vector wordt geïnjecteerd in het weefsel direct naast de verwijderde tumor en wordt alleen geïncorporeerd in delende cellen (met name de overgebleven tumorcellen). Vervolgens wordt de patiënt behandeld met een extended-release formulering van 5-fluorocytosine, een precursor van 5-fluorouracil die door de bloed-hersenbarrière heen kan. 5-fluorocytosine kan alleen door de CD-geïnfecteerde cellen worden omgezet in 5-fluorouracil. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date 2020 Expected Registration 2021 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Advanced therapy medicinal product (ATMP). Estimated Primary Completion Date fase II/III Toca 5 studie: december 2018. Therapeutic value Current treatment options PCV (procarbazine, CCNU (lomustine) en vincristine), temozolomide Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Oncoline; NCT02414165 Additional remarks Resection followed by administration of 4 mL Toca 511 (vocimagene amiretrorepvec). Toca 511 is administered by injection into the wall of the subject's tumor resection cavity on Day 1 (approximately 40 injections of 0.1 mL). Toca FC is an extended-release formulation of flucytosine. Toca FC will be administered at 220 mg/kg/day orally for 7-day courses beginning at least 6 weeks after resection and repeated approximately every 6 weeks. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Oncoline; Ho et al. Eur J Cancer. 2014 Sep;50(13):2309-18; Weller et al. Neuro Oncol. 2013 Jan; 15(1): 4–27 Additional remarks In 2011 werden er ongeveer 1.000 diagnoses hooggradig glioom gesteld. Bij ongeveer 70% van de gevallen vindt er resectie plaats (n=700). Ongeveer 90% van de gliomen keren terug (n=630). Expected cost per patient per year Cost References Additional remarks Kosten nog onbekend maar waarschijnlijk hoog. 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 Yes Indication extensions Metastatic solid tumors (CRC, RCC, Melanoma, Pancreatic, Lung & Breast) in fase I, newly diagnosed high grade glioma (preclinical) References Tocagen pipeline Additional remarks Other information Additional remarks