Olezarsen Medicine 04 June 2024 Extended indication Olezarsen monotherapy for add-on treatment of severe familial chylomicronemia syndrome in adults and elderly Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Olezarsen Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Unknown Extended indication Olezarsen monotherapy for add-on treatment of severe familial chylomicronemia syndrome in adults and elderly Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Ionis Portfolio holder Mechanism of action Antisense oligonucleotide Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Olezarsen is een antisense oligonucleotide, geconjugeerd met N-acetylgalactosamine (GalNAc), dat is ontworpen om de productie van door APOC3 gecodeerd apolipoproteïne C-III-eiwit te remmen. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date August 2024 Expected Registration September 2025 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks NCT04568434 Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks De effectiviteit lijkt gelijk aan volanesorsen, wat een 70% reductie in triglyceriden impliceert. Olezarsan heeft hoogstwaarschijnlijk niet de bijwerking thrombocytopenie. De fase 3 studie is in oktober 2023 afgerond, de resultaten moeten nog gepubliceerd worden. Expected patient volume per year Patient volume 18 - 180 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks Familiale chylomicronemie-syndroom (FCS) heeft een geschatte prevalentie van 1 op 100.000 en 1 op 1.000.000 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 Unknown Indication extensions References Additional remarks Other information Additional remarks