Mavorixafor Medicine 08 June 2021 Extended indication Xolremdi is indicated in patients 12 years of age and older for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Therapeutic value Possible added value Total cost Registration phase Registered Product Active substance Mavorixafor Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Xolremdi is indicated in patients 12 years of age and older for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Current proprietary name Already available biosimilars / generics Proprietary name Xolremdi 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 Normal trajectory Particularity ATMP No Submission date January 2025 Expected Registration April 2026 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in februari 2026 Therapeutic value Current treatment options De huidige behandeling is symptomatisch. Immunoglobuline vervangingstherapie en behandeling met antibiotica uit voorzorg kunnen infecties voorkomen. Therapeutic value Possible added value 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. Er wordt verwacht dat dit middel een belangrijke toevoeging zal zijn in de behandeling van dit zeldzame syndroom. Duration of treatment Frequency of administration 1 times a day Dosage per administration 400mg References NCT03995108; Expertopinie 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 het jaar 1990 en 2006 werd geschat op minder dan een 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 Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References adisinsight Additional remarks Other information Additional remarks