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Familial chylomicronemia syndrome (FCS)- Refile NDA for Volanesorsen (WAYLIVRA) with USFDA in H2 2020


Volanesorsen (WAYLIVRA) is the leading drug in Familial chylomicronemia syndrome (FCS) space globally., developed by Ionis & Akcea Therapeutics.

Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disease caused by mutations in the lipoprotein lipase, resulting in the accumulation of chylomicron in the plasma and hypertriglyceridaemia (elevated triglycerides, levels exceeding 1,000 mg / dL). Elevated triglycerides cause several complications in patients, the most serious being episodes of acute pancreatitis. 

FCS prevalence was 13 in 1,000,000 which is 13-fold higher than previously described as per new study by Vincent, et al, 2019

Severe HTG leading to Acute Pancreatitis

Key Facts

  • FCS is a rare inherited lipid disease that is often underdiagnosed and not managed.
  • There are currently no FDA approved treatments for FCS.
  • Management options mainly include adopting an extremely restricted and very low-fat diet, along with the avoidance of certain medications and alcohol.
    • The only universal long-term therapy is limiting total dietary fat intake to <10-15% of daily calories (15 to 20g per day).
    • Many patients have been treated with fibrates and statins with variable response, but many remain susceptible to pancreatitis.

Key Mellalta Meets Findings:

Volanesorsen (WAYLIVRA)- for Familial chylomicronemia syndrome (FCS)

Emerging treatments

Volanesorsen (WAYLIVRA)

Volanesorsen (WAYLIVRA) developed by Ionis & Akcea Therapeutics, is an antisense oligonucleotide (ASO) inhibitor of apoC3, which reduces TG levels by 70-80%, which has also been shown to reduce pancreatitis rates and improve well-being in the FCS and reduce TG and improve insulin resistance in FPLD. Volanesorsen received marketing authorisation in Europe in May 2019. The drug is also undergoing two trial studies i.e. Phase III trials (BALANCE) and Expanded Access study 

In 2018 Ionis Pharmaceuticals received a full response letter from the FDA for volanesorsen for the treatment of patients with familial chylomicronemia syndrome (FCS). There was no mention by Ionis/Akcea about the CRL, but it was likely due to the safety concern of thrombocytopenia (low platelets) associated with volanesorsen.

According to the recent release of the quarter, the company expects to refile the NDA for Volanesorsen (WAYLIVRA) in the United States with the FDA and also launch the drug in the additional EU countries.


ARO-APOC3 developed by Arrowhead, is designed to reduce the production of apolipoprotein C-III (apoC-III), a component of the triglyceride-rich lipoproteins (TRLs) including VLDL and chylomicrons.

The drug is currently undergoing Phase I study (NCT03783377) to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridaemia and familial chylomicronemia syndrome ( FCS).


Pradigastat (LCQ-908), a Novartis drug, is a potent, selective and orally active inhibitor of diacylglycerol acyltransferase 1 (DGAT1). The drug was being tested in Phase 3 in subjects with familial chylomicronemia (FCS) syndrome (HLP type I). The interim study results did not support the further extension of this study leading to its termination.

Mellalta Meets believes that, WAYLIVRA (volanesorsen) will be the first and only therapy for FCS, an extremely rare and life-threatening condition, to be available globally for an exceptionally long time with no short term competitors. 

Click here for more information on the direct and Indirect competitors for Elacestrant in ER+/HER2- mBC. If you wish to learn more on how Mellalta can be of assistance, Click here.

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