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Plozasiran

ARO-APOC3

Extended indication Familial chylomicronaemia syndrome in adults and elderly with elevated fasting triglycerides at scre
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Plozasiran
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Familial chylomicronaemia syndrome in adults and elderly with elevated fasting triglycerides at screening refractory to standard lipid lowering therapy, monotherapy
Proprietary name ARO-APOC3
Manufacturer Arrowhead
Mechanism of action Other, see general comments
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Additional remarks Apo C-III-remmer

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date February 2025
Expected Registration March 2026
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Indieningsdatum en verwachte registratie op basis van IHSI-inschatting.

Therapeutic value

Current treatment options Volanesorsen
Therapeutic value No estimate possible yet
Substantiation Van de Apo C-III-remmers is volanesorsen geregistreerd als behandeling voor familiale chylomicronemie-syndroom en is olezarsen in ontwikkeling. De angiopoietin-like protein 3 remmers vupanorsen, evinacumab en zodasiran zijn ook in ontwikkeling. De effectiviteit van plozasiran lijkt gelijk aan volanesorsen; in de SHASTA-2 studie werd een 57 tot 77% reductie in triglyceriden aangetoond. Plozasiran heeft hoogstwaarschijnlijk niet de bijwerking thrombocytopenie (1,3).
Frequency of administration 1 times every 12 weeks
References Spagnuolo & Hegele. Exp. Rev. Endocrin. & Metab. 2024 (1); NCT05089084 (PALISADE) (2); Gaudet et al. JAMA Cardio. 2024 (3).

Expected patient volume per year

Patient volume

18 - 180

Market share is generally not included unless otherwise stated.
References Orphanet. 2024 (1).
Additional remarks Familiale chylomicronemie-syndroom (FCS) heeft een geschatte prevalentie van 1 op 100.000 en 1 op 1.000.000 (1).

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

Indication extension Yes
Indication extensions Mixed dyslipidaemia (1); Severe hypertriglyceridaemia (2-5).
References NCT04998201 (MUIR) (1); NCT04720534 (SHASTA-2) (2); NCT06347003 (SHASTA-3) (3); NCT06347016 (SHASTA-4) (4); NCT06347133 (MUIR-3) (5).
Additional remarks Plozasiran zit in fase 2 voor mixed dyslipidaemia (1) en in fase 3-trials voor severe hypertriglyceridaemia (2-5). Op basis van een primary completion datum in juli 2026 wordt de severe hypertrigliceridaemia uitbreiding eind 2027 verwacht. De mixed dyslipidaemia uitbreiding wordt later verwacht.

Other information

There is currently no futher information available.