Extended indication Patients With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Mavorixafor
Domain Chronic immune diseases
Reason of inclusion New medicine (specialité)
Main indication Other chronic immune diseases
Extended indication Patients With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Manufacturer X4 Pharmaceuticals
Mechanism of action Other, see general comments
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional comments CXCR4 receptor antagonist; Cytotoxic T lymphocyte stimulant.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials
Additional comments Verwachte indieningsdatum op basis van primary completion datum fase 3 studie in september 2021.

Therapeutic value

Current treatment options De huidige behandeling is symptomatisch. Immunoglobuline vervangingstherapie en behandeling met antibiotica uit voorzorg kunnen infecties voorkomen.
Therapeutic value No estimate possible yet
Substantiation Er is onderzoek gedaan in een groep van 8 patiënten, de maximale follow-up was 28,6 maanden. Er werd herstel van het aantal neutrofiele granulocyten en lymfocyten gezien, 75% reductie van verruca vulgaris en reductie van infecties bij behandeling langer dan 6 maanden.
Duration of treatment continuous
Frequency of administration 1 times a day
Dosage per administration 400 mg
References NCT03995108
Additional comments 4 capsules van 100mg.

Expected patient volume per year

Patient volume

5

Market share is generally not included unless otherwise stated.

References Orpha.net
Additional comments WHIM-syndroom is extreem zeldzaam. De incidentie in Frankrijk tussen 1990 en 2006 werd geschat op minder dan 1/4.000.000 geboortes.

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

Indication extension No
References adisinsight

Other information

There is currently no futher information available.