Mavorixafor Medicine 08 June 2021 Extended indication Patients With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending 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. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer X4 Pharmaceuticals Portfolio holder Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks CXCR4 receptor antagonist; Cytotoxic T lymphocyte stimulant. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date January 2025 Expected Registration February 2026 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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 This assessment does not indicate any potential inclusion in the package. Substantiation Er is onderzoek gedaan in een groep van acht 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 zes maanden. Duration of treatment Frequency of administration 1 times a day Dosage per administration 400mg References NCT03995108 Additional remarks 4 capsules van 100mg. Expected patient volume per year Patient volume 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orpha.net Additional remarks WHIM-syndroom is extreem zeldzaam. De incidentie in Frankrijk tussen 1990 en 2006 werd geschat op minder dan 1 op de 4.000.000 geboortes. 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 No Indication extensions References adisinsight Additional remarks Other information Additional remarks