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Olezarsen

Extended indication Olezarsen monotherapy for add-on treatment of severe familial chylomicronemia syndrome in adults and
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Olezarsen
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Extended indication Olezarsen monotherapy for add-on treatment of severe familial chylomicronemia syndrome in adults and elderly
Manufacturer Ionis
Mechanism of action Antisense oligonucleotide
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
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
ATMP No
Submission date August 2024
Expected Registration September 2025
Orphan drug No
Registration phase Registration application pending
Additional remarks NCT04568434

Therapeutic value

Therapeutic value No estimate possible yet
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.
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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.