Bidridistrogene xeboparvovec Medicine 08 June 2021 Extended indication Patients With Limb-girdle muscular dystrophy type 2E (LGMD2E) (Beta-sarcoglycan Deficiency) aged 4 to 15. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Bidridistrogene xeboparvovec Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Muscular diseases other Extended indication Patients With Limb-girdle muscular dystrophy type 2E (LGMD2E) (Beta-sarcoglycan Deficiency) aged 4 to 15. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sarepta Portfolio holder Mechanism of action Gene therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2023 Expected Registration 2024 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Nog geen Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De eerste resultaten uit de fase 2 studie bij kinderen (n=6) beschrijft na 2 jaar een toename van rond de 50% eiwitexpressie in de spieren en een verbetering in timed test en NSAD score. Verdere resultaten dienen afgewacht te worden om te zien of er een echte meerwaarde is voor de patiënt. Duration of treatment Frequency of administration Dosage per administration References NCT03652259 Additional remarks Patients with diagnosis of LGMD2E will receive SRP-9.003 administered through a single systemic injection. Expected patient volume per year Patient volume 1 - 9 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Opha.net Additional remarks De prevalentie van deze deficiëntie is 1-9 op de 1.000.000. Enkel kinderen van 4 tot 15 komen in aanmerking. Dit zou betekenen dat er mogelijk ook 1 tot 9 patiënten in Nederland in aanmerking zullen komen aangezien er 1 miljoen inwoners in Nederland zijn binnen deze leeftijdsgroep. 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 Adis Insight Additional remarks Other information Additional remarks