Waylivra

Balancing the Challenges of FCS

Waylivra is the only therapy licensed* to treat familial chylomicronemia syndrome (FCS), as an adjunct to diet in adult patients with genetically confirmed disease and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate (1)
What is FCS?

Balancing the Challenges of FCS

Waylivra is the only therapy licensed* to treat familial chylomicronemia syndrome (FCS), as an adjunct to diet in adult patients with genetically confirmed disease and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate (1)
What is FCS?

Waylivra is the only therapy licensed to treat familial chylomicronemia syndrome (FCS)1*

 

FCS is a debilitating disease with a high risk of pancreatitis due to high triglyceride (TG) levels over a prolonged time.2,3

Observational data suggest that the incidence of acute pancreatitis increases 3% for every increment of 100 mg/dl in TG levels > 1000 mg/dl; therefore a decrease in TG levels to any extent below this threshold may result in a decrease in the risk of acute pancreatitis.4

In the APPROACH study, Waylivra significantly reduced mean fasting TG levels by 77% at 3 months in patients with FCS. The incidence of pancreatitis was numerically lower in the Waylivra group compared with placebo1,4

Results from the open label extension (OLE) study** showed sustained reduction in plasma TG levels up to 156 weeks*** with no new safety signals. These results support the long-term use of Waylivra in patients with FCS.5

With proactive monitoring, Waylivra has a manageable safety profile5

Waylivra is a self-administered injection delivered in a single-use prefilled syringe1

Waylivra

*by EMA

**This study had some limitations, including those inherent with an open-label study design. Without a placebo or non-treatment group, assessing whether an actual. Reduction in acute pancreatitis risk occurred is challenging.

***Total number of weeks from both APPROACH and COMPASS study.

References

  1. Waylivra® SmPC, Nov 2022.
  2. Gaudet D, Brisson D, T remblay K, Alexander VJ, Singleton W, Hughes SG, Geary RS, Baker BF, Graham MJ, Crooke RM, Witztum JL. Targeting APOC3 in the familial chylomicronemia syndrome. N Engl J Med. 2014 Dec 4;371(23):2200-6. doi: 10.1056/NEJMoa1400284. PMID: 25470695.
  3. Davidson M, Stevenson M, Hsieh A, Ahmad Z, Roeters van Lennep J, Crowson C, Witztum JL. The burden of familial chylomicronemia syndrome: Results from the global IN-FOCUS study. J Clin Lipidol. 2018 Jul-Aug;12(4):898-907.e2. doi: 10.1016/j.jacl.2018.04.009. Epub 2018 Apr 26. PMID: 29784572.
  4. Witztum JL et al. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019 Aug 8;381(6):531-542. doi: 10.1056/NEJMoa1715944. PMID: 31390500.
  5. Witzum JL et al., Journal of Clinical Lipidology (2023) 000, 1–14 Published: March 21, 2023. DOI:https://doi.org/10.1016/j.jacl.2023.03.007