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


Manufacturer Rocket
Mechanism of action Gene therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the therapeutic lentiviral vector, Chim-CD18-WPRE, RP-L201


Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Particularity New medicine with Priority Medicines (PRIME)
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials

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
Duration of treatment one-off
Frequency of administration 1 times a day
References NCT03812263

Expected patient volume per year

Patient volume

< 5

Market share is generally not included unless otherwise stated.

References https://ghr.nlm.nih.gov/condition/leukocyte-adhesion-deficiency-type-1#statistics
Additional comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.