Larotrectinib Medicine 12 June 2018 Extended indication Solid tumours - with NTRK1, NTRK2 or NTRK3 gene fusion. Therapeutic value Possible added value Total cost Registration phase Clinical trials Product Active substance Larotrectinib Domain Oncology and Hematology Reason of inclusion Main indication Oncology other Extended indication Solid tumours - with NTRK1, NTRK2 or NTRK3 gene fusion. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Bayer Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Locally advanced or metastatic solid tumors with NTRK gene fusion in adult and pediatric patients. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine ATMP Unknown Submission date 2018 Expected Registration May 2019 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks In de VS wel een orphan drug status. Therapeutic value Current treatment options Afhankelijk van het tumortype. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation De Fase I/II studie van Drilon et al. laat veelbelovende resultaten zien. Duration of treatment Frequency of administration Dosage per administration References Drilon et al. N Engl J Med 2018; 378:731-739; NCT02637687; NCT02576431 Additional remarks Fase I voor volwassen patiënten, fase 1/2 voor pediatrische patiënten (SCOUT), fase 2 voor volwassenen en adolescenten (NAVIGATE). Expected patient volume per year Patient volume 100 - 300 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Stransky et al. Nat Commun. 2014 Sep 10; 5: 4846 Additional remarks Er worden per jaar ongeveer 100.000 solide tumoren gediagnosticeerd. Incidentie van NTKR fusies bedraagt ongeveer 0,3%. Dit zou een populatie van 300 patiënten bedragen met een NTKR-fusie. De werkgroep schat het patiëntvolume in op ongeveer 100. 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 Unknown Indication extensions References Additional remarks Other information Additional remarks