Lomitapide Medicine 04 June 2024 Extended indication Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) Therapeutic value Possible benefit in ease of use Total cost € 7,740,000.00 Registration phase Clinical trials Product Active substance Lomitapide Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Lipid-lowering medications Extended indication Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) Current proprietary name Already available biosimilars / generics Proprietary name Lojuxta Manufacturer Amryt Portfolio holder Mechanism of action Unknown Route of administration Unknown Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2024 Expected Registration 2025 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Evinacumab Therapeutic value Possible benefit in ease of use This assessment does not indicate any potential inclusion in the package. Substantiation Lomitapide heeft orale toediening (tabletten). Voor kinderen met HoFH met naaldenangst of die ver weg wonen van expertise centrum kan orale toediening een voordeel zijn ten opzichte van evinacumab dat 1 keer per maand intraveneus wordt toegediend. Duration of treatment Frequency of administration 1 times a day Dosage per administration 20 - 60mg References NCT04681170 Additional remarks Age 5-10 years Lomitapide dosing will commence with 2mg at week 1 for 8 Weeks,then increase to 5mg Week 8±3 days, 10 mg at Week 12±3 days to the maximum allowable dose of 20 mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values Intervention: Drug: Lomitapide Age 11-15years Lomitapide dosing will commence with 2mg at week 1 for 4 Weeks, then increase to 5mg Week 4±3 days, 10 mg at Week 8±3 days,20mgs at week, 12±3 days to the maximum allowable dose of 40 mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values Intervention: Drug: Lomitapide 16 to ≤17 years, Lomitapide dosing will commence with 5mg at week 1 for 4 Weeks, then increase to 10mg Week 4±3 days,20 mg at Week 8±3 days,40mgs at week, 12±3 days to the maximum allowable dose of 60 mg by Week 16±3 days or the MTD by Week 20±3 days based upon acceptable safety and tolerability criteria in addition to LDL C values Intervention: Drug: Lomitapide Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Record Evinacumab met indicatie: Evkeeza is indicated as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH). Additional remarks Homozygote FH is een zeldzame aandoening. Er zijn ongeveer 50 patiënten in Nederland. Niet alle patiënten komen in aanmerking voor deze behandeling gezien zij uit zullen komen met andere behandelingen. Geschat aantal in Nederland is 10 patiënten. Expected cost per patient per year Cost € < 774,000.00 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 medicijnkosten.nl Additional remarks Voor de volwassen patiënten is er een financieel arrangement afgesloten. Lojuxta capsule 20mg Lomitapide Amryt pharmaceuticals dac € 776,86 per stuk vergoed. Bij een maximale dosis betekent dit dat de kosten jaarlijks €774.000 per patiënt zijn. Potential total cost per year Total cost € 7,740,000.00 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