RP-L201 Medicine 10 June 2020 Extended indication Patients With Leukocyte Adhesion Deficiency-I of 3 Months and older Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance RP-L201 Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Patients With Leukocyte Adhesion Deficiency-I of 3 Months and older Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Rocket Portfolio holder Mechanism of action Gene therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201 Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Symptomatische behandelingen zijn onder andere hygiënische maatregelen, levenslange antibiotische profylaxe en tijdige en adequate behandeling van infecties. De enige genezende behandeling is een beenmergtransplantatie. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT03812263 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 https://ghr.nlm.nih.gov/condition/leukocyte-adhesion-deficiency-type-1#statistics Additional remarks Er zijn mondiaal tenminste 300 gevallen van deze ziekte bekend. Het patiëntvolume in Nederland zal niet meer dan vijf patiënten bedragen. 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