Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News
  • Published:

Phase 3 data for PCSK9 inhibitor wows

Nature Biotechnology volume 31, pages 1057–1058 (2013)Cite this article

Subjects

Access through your institution
Buy or subscribe

Pfizer has hinted that it may wait for outcomes data before seeking approval, but both the Regeneron and Sanofi partnership and Amgen plan to file for approval before completing their outcomes studies. “It's been our assessment that we're going to file based on the LDL-lowering data that we have,” says Scott Wasserman, global development lead for cardiology at Amgen. “We're looking for a global rollout in 2016 for lipid-lowering,” says Jay Edelberg, head of Sanofi's PCSK9 development and launch unit. Any approval based on surrogate data would probably be limited to patients in high-risk categories. “Initially you could approve them for familial hypercholesterolemia, for example, and for people who are completely statin-intolerant and who have had a heart attack,” says John Kastelein, who chairs the department of vascular medicine at the Academic Medicine Center, of the University of Amsterdam, in The Netherlands.

Some critics have urged caution, pointing to the failure of other drugs, such as Merck's approval of cholesterol absorption inhibitor Zetia (ezetimibe), to deliver improved cardiovascular outcomes despite their ability to lower cholesterol. George Yancopoulos, Regeneron's founding scientist and CSO, says the effect on LDL-c has to be significant in order to translate into long-term cardiovascular benefit. “If you don't lower cholesterol enough you're not going to have a big enough effect,” he says. In the recently reported phase 3 monotherapy trial, hypercholesterolemia patients on alirocumab achieved a mean reduction from baseline in LDL-c levels of 47.2% at 24 weeks, whereas those on the comparator drug Zetia (which, in the real world, is usually used in combination with a statin), achieved a mean reduction of 15.6%. The result was widely expected, but the absence of any signs of resistance to therapy due to antibody-associated immunogenicity was “reassuring,” Kastelein says.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Access through your institution

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Author information

Authors and Affiliations

  1. Dublin

    Cormac Sheridan

Authors
  1. Cormac Sheridan
    View author publications

    Search author on:PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Sheridan, C. Phase 3 data for PCSK9 inhibitor wows. Nat Biotechnol 31, 1057–1058 (2013). https://doi.org/10.1038/nbt1213-1057

Download citation

  • Published:

  • Issue date:

  • DOI: https://doi.org/10.1038/nbt1213-1057

This article is cited by

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing