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Waylivra™ (volanesorsen), first therapy for familial chylomicronemia syndrome
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Waylivra™ (volanesorsen), first therapy for familial chylomicronemia syndrome

Waylivra™ (volanesorsen),

first therapy for familial chylomicronemia syndrome

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization for Waylivra™ (volanesorsen) for the treatment of familial chylomicronemia syndrome (FCS). This antisense oligonucleotide developped by Ionis Pharmaceuticals and its affiliate Akcea Therapeutics is intented to be used as an adjunct to diet in adult patients with genetically confirmed FCS who are at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. There is no effective therapy for FCS currently available. This ultra-rare disease is caused by impaired function of the enzyme lipoprotein lipase (LPL). It is characterized by severe hypertriglyceridemia (>880mg/dL) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic diabetes.

Triglycerides reduced by 77%
Volanesorsen has been designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides. The CHMP’s positive opinion is based on results from the APPROACH study and its ongoing open label extension study. Results from this phase 3 program show hat in comparison to placebo, treatment with Waylivra™ reduced triglycerides by 77 percent after 3 months of treatment. The most common adverse events in the study were injection site reactions and reductions in platelet levels. Waylivra™ is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy (FPL). Akcea anticipates reporting top-line data from this study in mid-2019.

Second approval for Akcea
Waylivra™ will be the second medicine for which Akcea Therapeutics has received approval. The company has already got US and EU green light for Tegsedi® (inotersen). This antisense oligonucleotide is designed to reduce the production of transthyretin and it is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR).

“We will build on the strong infrastructure we have in place for Tegsedi® in Europe as we prepare for the launch of Waylivra™,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “This is a testament to the European Regulatory Authorities’ commitment to facilitating access to innovative medicines for patients in need.”Akcea Therapeutics is also advancing a mature pipeline of novel drugs for the treatment of cardiometabolic lipid disorders. These antisense nucleotides include AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx which are developed in partnership with Novartis for the treatment of hyperlipoproteinemia (a) and hypertriglyceridemia at significant risk of cardiovascular disease. The two other products under development, AKCEA-ANGPTL3 and AKCEA-TTR-LRx, are intented respectively for the treatment of patients with both hereditary and wild-type forms of transthyretin amyloidosis and for the treatment of nonalcoholic fatty liver disease (NAFLD) with metabolic complications that include hypertriglyceridemia, type 2 diabetes or nonalcoholic steatohepatitis (NASH).


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