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Long-term effects of volanesorsen on triglycerides and pancreatitis in patients with familial chylomicronaemia syndrome (FCS) in the UK Early Access to Medicines Scheme (EAMS)

Lookup NU author(s): Dr Fiona Jenkinson

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Abstract

© 2023 Elsevier B.V.Background and aims: The VOL4002 study assessed the efficacy and safety of volanesorsen in 22 adults with genetically confirmed familial chylomicronaemia syndrome (FCS) treated in the UK Early Access to Medicines Scheme (EAMS), with (“prior exposure”) or without (“treatment naive”) previous treatment in the APPROACH and/or APPROACH-OLE volanesorsen phase 3 studies. Methods: Data collection focused on triglyceride (TG) levels, platelet counts and pancreatitis events. Pancreatitis incidence during volanesorsen treatment was compared against the 5-year period preceding volanesorsen exposure. Volanesorsen 285 mg was self-administered subcutaneously once every 2 weeks. Results: Individual patient volanesorsen exposure ranged from 6 to 51 months (total cumulative exposure, 589 months). Among treatment-naive patients (n = 12), volanesorsen treatment resulted in an averaged median 52% reduction (−10.6 mmol/L) from baseline (26.4 mmol/L) in TG levels at 3 months, which were maintained through time points over 15 months of treatment (47%–55% reductions). Similarly, prior-exposure patients (n = 10) experienced a 51% reduction (−17.8 mmol/L) from pre-treatment baseline (28.0 mmol/L), with reductions of 10%–38% over 21 months of treatment. A comparison of pancreatitis event rates found a 74% reduction from the 5-year period before (one event/2.8 years) and during (one event/11.0 years) volanesorsen treatment. Platelet declines were consistent with observations in phase 3 clinical trials. No patient recorded a platelet count <50 × 109/L. Conclusions: This longitudinal study supports the efficacy of volanesorsen in patients with FCS for lowering TG levels over treatment periods up to 51 months with no apparent safety signals related to increased duration of exposure.

Publication metadata

Author(s): Jones A, Peers K, Wierzbicki AS, Ramachandran R, Mansfield M, Dawson C, Ochoa-Ferraro A, Soran H, Jenkinson F, McDowell I, Downie P, Hamilton P, Jones RD

Publication type: Article

Publication status: Published

Journal: Atherosclerosis

Year: 2023

Volume: 375

Pages: 67-74

Print publication date: 01/06/2023

Online publication date: 15/05/2023

Acceptance date: 09/05/2023

ISSN (print): 0021-9150

ISSN (electronic): 1879-1484

Publisher: Elsevier Ireland Ltd

URL: https://doi.org/10.1016/j.atherosclerosis.2023.05.008

DOI: 10.1016/j.atherosclerosis.2023.05.008

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