Volanesorsen: First Global Approval
Julia Paik
Sean Duggan
10.6084/m9.figshare.8637239.v1
https://adisjournals.figshare.com/articles/online_resource/Volanesorsen_First_Global_Approval/8637239
<p>Compliance with Ethical Standards</p><p><b>Funding</b> The preparation of this review was not supported by any external funding.</p><p><b>Conflicts of interest</b><b><i> </i></b><b> </b>During
the peer review process the manufacturer of the agent under review was offered
an opportunity to comment on the article. Changes resulting from any comments
received were made by the authors on the basis of scientific completeness and
accuracy. Julia Paik and Sean Duggan are salaried employees of Adis
International Ltd/Springer Nature, are responsible for the article content and declare no relevant conflicts
of interest.</p><p>
<br></p><p>Additional information about this Adis Drug Review can be found <a href="http://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews"><strong>here</strong></a></p><p><br></p><p><strong>Abstract</strong></p><p>Volanesorsen (Waylivra<sup>®</sup>), an antisense oligonucleotide
inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis
Pharmaceuticals through its subsidiary company, Akcea Therapeutics, to treat
familial chylomicronemia syndrome (FCS), hypertriglyceridemia and familial
partial lipodystrophy (FPL). In May 2019, volanesorsen was approved in the EU for
the treatment of adult patients with FCS based on positive results from the
multinational, phase III APPROACH and COMPASS studies. Other clinical
trials are ongoing to assess its utility in hypertriglyceridemia, FPL and
partial lipodystrophy. This article summarizes the milestones in the development
of volanesorsen leading to this first approval as an adjunct to diet in adult
patients with genetically confirmed FCS and at high risk for pancreatitis, in
whom response to diet and triglyceride lowering therapy has been inadequate.</p><p>
<br></p><p>© Springer Nature Switzerland AG 2019</p>
2019-07-29 21:10:27
Volanesorsen
Waylivra
Familial Chylomicronemia Syndrome (FCS)
Hypertriglyceridemia
Familial partial lipodystrophy
chylomicronemia
Adis Insight Report