Akcea Therapeutics and Ionis Pharmaceuticals have announced that Waylivra (volanesorsen) has received conditional marketing authorisation from the European Commission as an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.1
This authorisation follows the positive opinion recommendation provided by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).2 As part of the conditional marketing authorisation, Akcea and Ionis will conduct a non-interventional post-authorisation safety study (PASS) based on a Registry.
“Volanesorsen is the only authorised treatment for people with FCS and is a major milestone for the global FCS community. This also marks Akcea’s second drug authorisation in the last year,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “We are very grateful to all the patients and physicians around the world who participated in our clinical trials. Their insights about the challenges of this devastating disease and their stories of how they have benefited from volanesorsen continue to motivate us.”
FCS is an ultra-rare debilitating disease, caused by impaired function of the enzyme, lipoprotein lipase (LPL).3 It results in significant risk and disease burden, including unpredictable and potentially fatal acute pancreatitis as well as chronic complications due to permanent organ damage.3
“Volanesorsen is the only treatment available for patients with FCS which makes this authorisation a landmark event for the global FCS community. Patients, their caretakers and their families have been suffering without any therapeutic option. Now patients across Europe can access a medicine that may help address their severely elevated triglycerides which can give them an option to help manage their condition. High triglycerides can lead to a multitude of severe and daily chronic symptoms such as abdominal pain and increased risk of pancreatitis which have a significant daily impact on people living with FCS,” said Jill Prawer, founder and chair, LPLD Alliance.
Volanesorsen is an antisense oligonucleotide drug designed by Ionis and co-developed by Akcea and Ionis to reduce the production of ApoC-III, a protein that regulates plasma triglycerides.1 Volanesorsen is a self-administered, subcutaneous injection in a single-use, prefilled syringe.1
The EC’s marketing authorisation of volanesorsen is based on results from the Phase III APPROACH study4 and the ongoing APPROACH Open Label Extension study5 and is supported by results from the Phase III COMPASS study.6 Results from the Phase III APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with volanesorsen reduced triglycerides by 77% after 3 months of treatment.1,4 The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels.1,4 In addition to the open label extension study, there are also ongoing global Early Access Programmes for volanesorsen.7
European Medicines Agency. Waylivra Summary of Product Characteristics. Available at: www.ema.europa.eu/en. Last accessed May 2019.
European Medicines Agency. Waylivra (volanesorsen) Summary of opinion (initial authorisation). Available at: www.ema.europa.eu/en/documents/smop-initial/chmp-summary-positive-opinio…. Last accessed May 2019.
Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011.
Gaudet D, et al. (2017) The APPROACH Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS). Journal of Clinical Lipidology; 11(3):814-5.
ClinicalTrials.gov. The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome. Available at: https://clinicaltrials.gov/ct2/show/NCT02658175. Last accessed May 2019.
ClinicalTrials.gov. The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia. Available at: https://clinicaltrials.gov/ct2/show/NCT02300233. Last accessed May 2019.
ClinicalTrials.gov. Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS). Available at: https://clinicaltrials.gov/ct2/show/NCT03544060. Last accessed May 2019.