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Volanesorsen: First Global Approval

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Abstract

Volanesorsen (Waylivra®), 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.

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Authors and Affiliations

  1. Springer Nature, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand

    Julia Paik & Sean Duggan

Authors
  1. Julia Paik
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  2. Sean Duggan
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Correspondence to Julia Paik.

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Funding

The preparation of this review was not supported by any external funding.

Conflict of interest

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.

Additional information

Additional information for this AdisInsight Report can be found at https://doi.org/10.6084/m9.figshare.8637239.

This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Paik, J., Duggan, S. Volanesorsen: First Global Approval. Drugs 79, 1349–1354 (2019). https://doi.org/10.1007/s40265-019-01168-z

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