RP-L201

Active substance
RP-L201
Domain
Chronic immune diseases
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Other chronic immune diseases
Extended indication
Patients With Leukocyte Adhesion Deficiency-I of 3 Months and older

1. Product

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

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Accelerated assessment
Particularity
New medicine with Priority Medicines (PRIME)
Submission date
2020
Expected Registration
2021
Orphan drug
Yes
Registration phase
Clinical trials
Additional comments
Dit geneesmiddel is geclassificeerd als een ATMP.

3. 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
Frequency of administration
1 times a day
References
NCT03812263

4. 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.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

There is currently nothing known about indication extensions.

9. Other information

There is currently no futher information available.