Forthcoming Expert Guideline May Upgrade PCSK9 Inhibitors

–Results of the FOURIER trial might bolster the embattled cholesterol drugs.

An updated expert statement may provide new support for expanded use of PCSK9 inhibitor cholesterol drugs.

Since their initial approval two years ago usage of the expensive new drugs (Repatha from Amgen and Praluent from Sanofi and Regeneron) has been severely limited due to strict restrictions on reimbursement. Lack of evidence showing long term benefits of the drugs has been the main roadblock. The recent publication of the positive (but not overwhelmingly convincing) FOURIER outcomes trial with Repatha has sparked hope (by advocates) and fear (by critics) that restrictions on usage will begin to be eased.

Now an early draft of a new version of an expert consensus statement from the American College of Cardiology may indicate new support for the controversial new drugs. The current version of the statement, the ACC Expert Consensus Decision Pathway on the Role of Non-Statin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk, which was updated last year, places PCSK9 inhibitors third in line after statins and ezetimibe (Zetia) for the treatment of people with atherosclerotic cardiovascular disease. The major change in the new draft is the equal treatment of PCSK9 inhibitors and ezetimibe.

One Wall Street analyst, Cowen’s Eric Schmidt, said the early draft of the statement is “quite favorable to Amgen.” He said the statement “would support the use of anti-PCSK9 therapy in a much broader patient population.”

However, the updated statement has not been written in stone. The ACC received 384 comments in response to the draft, which was also subject to internal review. The first author of the expert consensus statement, Donald Lloyd-Jones (Northwestern University) said “that it has changed significantly since that draft,” though the details of the revised version are embargoed. He said the final published document is expected to be published in September.

“I wouldn’t read too much into this draft,” he added.

Lloyd-Jones also emphasized the differences between an expert consensus document and the more formal guidelines, which the ACC produces in conjunction with the American Heart Association (AHA). “The formal guidelines for cholesterol management are underway and will deal with the formal evidence review and make formal evidence-based recommendations. This document is a bridge to that process,” said Lloyd-Jones. Sidney Smith (UNC) said that the updated formal guideline on cholesterol lowering has been started but is not expected to be published until 2018.

Sanjay Kaul (Cedars-Sinai) asked “why the rush to update the statement prior to the FDA approval of the expanded label for evolocumab?” Lloyd-Jones said that the “ACC felt it important to update the document in response to the FOURIER results in particular. I am unaware of FDA’s timeline.”


  1. JDPatten says

    Cholesterol: friend of the Elderly.
    You may be cranky about the particular authors of the following article – and you might have cause – but I assure you that, as one of this coterie, I’ll not be reducing my LDL until further RCTs definitively disprove their work.

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