Beremagene geperpavec Medicine 08 December 2020 Extended indication Dominant and recessive dystrophic epidermolysis bullosa (DEB) Therapeutic value Possible added value Total cost € 85,702,000.00 Registration phase Clinical trials Product Active substance Beremagene geperpavec Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Skin diseases Extended indication Dominant and recessive dystrophic epidermolysis bullosa (DEB) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Krystal Biotech Portfolio holder Mechanism of action Gene therapy Route of administration Local Therapeutical formulation Gel Budgetting framework Unknown Centre of expertise Additional remarks Beremagene geperpavec (ook bekend als Bercolagene telserpavec of KB103) is een niet-integrerend, replicatie-incompetente HSV-1 wat het humane collageen VII eiwit tot expressie brengt. Het bekostigingskader is nog niet bekend. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date 2024 Expected Registration 2025 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Palliatieve zorg Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er is momenteel geen behandeling voorhanden die de aandoening kan genezen. Duration of treatment Frequency of administration Dosage per administration References NCT04491604 Additional remarks Het betreft hier een doorlopende behandeling, de toedieningsfrequentie is nog niet bekend. Expected patient volume per year Patient volume < 146 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Afdeling Dermatologie, UMCG Additional remarks In het UMC-Groningen worden momenteel alle patiënten voor deze aandoening behandeld. In 2020 waren er 146 patiënten met DEB (dominant en recessief) bekend. Expected cost per patient per year Cost € 587,000.00 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://antonhealth.com/fda-approves-new-topical-gene-tx-vyjuvek/ Additional remarks De prijs bedraagt $24,250 per dosis in de Verenigde Staten. Dit betekent grofweg dat een jaarbehandeling $631,000 per patiënt per jaar zal kosten. De prijs in Nederland is nog niet bekend. Potential total cost per year Total cost € 85,702,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks