Olezarsen

Extended indication	Familial chylomicronemia syndrome
Therapeutic value	No estimate possible yet
Registration phase	Clinical trials

Product

Active substance	Olezarsen
Domain	Metabolism and Endocrinology
Reason of inclusion	New medicine (specialité)
Extended indication	Familial chylomicronemia syndrome
Manufacturer	Ionis
Route of administration	Subcutaneous
Therapeutical formulation	Injection
Budgetting framework	Extramural (GVS)
Additional remarks	Olezarsen is a next-generation, N-acetylgalactosamine (GalNAc)–conjugated, antisense oligonucleotide designed to inhibit APOC3–encoded apolipoprotein C-III protein production.

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.

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 1:100.000 en 1: 1000.000

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 1:100.000 en 1: 1000.000

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.