Marnetegragene autotemcel

Extended indication	Patients With Leukocyte Adhesion Deficiency-I of 3 Months and older
Therapeutic value	No estimate possible yet
Registration phase	Clinical trials

Product

Active substance	Marnetegragene autotemcel
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 remarks	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)
ATMP	Yes
Submission date	June 2023
Expected Registration	March 2024
Orphan drug	Yes
Registration phase	Clinical trials

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

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 remarks	Er zijn mondiaal tenminste 300 gevallen van deze ziekte bekend. Het patiëntvolume in Nederland zal niet meer dan vijf patiënten bedragen.

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 remarks

Er zijn mondiaal tenminste 300 gevallen van deze ziekte bekend. Het patiëntvolume in Nederland zal niet meer dan vijf patiënten bedragen.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.