Vosoritide Medicine 06 June 2023 Extended indication Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. Therapeutic value No estimate possible yet Total cost € 1,575,000.00 Registration phase Registered Product Active substance Vosoritide Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Other metabolism and Endocrinology Extended indication Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. Current proprietary name Already available biosimilars / generics Proprietary name Voxzogo Manufacturer BioMarin Portfolio holder BioMarin Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise UMC Utrecht Skeletal Dysplasia unit. Erasmus Rotterdam(Toekomstig). Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date December 2022 Expected Registration October 2023 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in september 2023. Therapeutic value Current treatment options Op dit moment is er geen behandeling voor achondroplasia. Vosoritide is wel geregistreerd voor patiënten ouder dan 2 jaar. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Mogelijk is er meer winst als er al onder de twee jaar begonnen wordt met de behandeling en daardoor mogelijk een gunstigere skeletgroei. Daarnaast is er mogelijk een gunstig effect op het voorkomen van vernauwing van het wervelkanaal. Duration of treatment Frequency of administration 1 times a day Dosage per administration 15·0 μg/kg References Klinisch genetica NL, Lancet September 05, 2020, Genetics in Medicine volume 23, pages 2443–2447 (2021) EMA web site Additional remarks Gemiddelde eindlengte is voor mannen 1 meter 31, voor vrouwen 1 meter 24. Vosoritide geeft groei van 1.57cm per jaar en verbetering van lichaamsproporties. Onderzocht in leeftijdsgroep 5 tot 17 jaar. Beste effect op groei tussen 2 tot 5 jaar. De vraag is of dat het middel ook goed werkt tijdens groeispurt omdat deze afwijkend is bij achondroplasie. Expected patient volume per year Patient volume 7 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Birth prevalence of achondroplasia: A systematic literature review and meta‐analysis. Pamela K. Foreman. Am J Med Genet A. 2020 (1); Additional remarks De geboorteprevalentie is 5 op de 100.000. Dit betekent dat er 7 nieuwe patiënten per jaar in aanmerking komen (1). Expected cost per patient per year Cost € 200,000.00 - 250,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References https://www.bvkm.nl/thema/achondroplasie-en-vosoritide-veelgestelde-vragen-en-antwoorden/ Additional remarks De fabrikant heeft in eerste instantie ingezet op gemiddeld €200.000 tot € 250.000 per patiënt per jaar. Potential total cost per year Total cost € 1,575,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks