WAYLIVRA PASS
Research type
Research Study
Full title
WAYLIVRA® Post-Authorisation Safety Study (PASS) and Product Registry
IRAS ID
289319
Contact name
Handrean Soran
Contact email
Sponsor organisation
Akcea Therapeutics Ireland Limited
Duration of Study in the UK
15 years, 0 months, 1 days
Research summary
Familial Chylomicronaemia Syndrome (FCS) is a serious, rare disorder of lipid metabolism affecting an estimated 3,000 to 5,000 patients globally. Characterised by extremely high serum triglycerides, patients with FCS have several severe and potentially life-threatening complications associated with the disease, including acute pancreatitis, which can be fatal or lead to pancreatic damage. There is a high unmet need for novel, effective therapies to treat patients with FCS.
The aim of this study (PASS- and registry phase) is to further characterise the safety and effectiveness of WAYLIVRA (Compound: Volanesorsen sodium) in patients with FCS under real-world conditions.
This study will be conducted in two phases. The first phase is being conducted as part of the pharmacovigilance plan agreed with the European Medicines Agency (EMA) in the European Risk Management Plan (RMP) and is designated as a Category 2 PASS. The first phase will be concluded after a study term of 5 years in
which at least data on 247 person-years of exposure in patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate have been collected. Following this initial phase, the study will continue as the WAYLIVRA Product Registry for the
commercial life of the drug, to obtain long-term data on safety and efficacy of WAYLIVRA.Adult patients (≥ 18-years-old) prescribed WAYLIVRA.
Study contains of a PASS study, which ends in 2025 (study report in 2026) and a Registry that end on 1Nov2035 (study report in 2036)
Volanesorsen sodium is registered by EMA in 2019, on condition that the Sponsor performs a PASS which is carried out over a period of 5 years (with 247 patient years) EMEA/H/C/PSP/S/0080
This is an observational safety study, and no experimental or extra treatments are done. All participants in this study will receive WAYLIVRA according to the usual clinical practice followed by their prescribing physician.
Approximately 200 patients from France, Germany, Italy, The Netherlands, Spain, Sweden, UK and other European countries will participate in this study.REC name
London - City & East Research Ethics Committee
REC reference
21/PR/0299
Date of REC Opinion
26 Mar 2021
REC opinion
Further Information Favourable Opinion