No, CRISPR gene editing technology is not going to “cure” heart disease. But a New York Times story by Gina Kolata on an extremely early study in animals prominently plays up just this extremely unlikely claim. The Times story is based on a press release issued by Verve Therapeutics, a new biotechnology company founded by Sekar Kathiresan, an influential cardiologist and genomic…
The ODYSSEY Trial Ends Well— But Will It Be Enough?
March 10, 2018 by 8 Comments
Sanofi and Regeneron achieved a modest victory with the much anticipated ODYSSEY Outcomes trial of its cholesterol lowering drug Praluent (alirocumab). The trial met its primary endpoint and even reported a significant improvement in all-cause mortality. But the mortality finding has an asterisk attached to it and it is far from clear whether the overall…
Moment Of Truth For Struggling And Expensive Cholesterol Drugs
February 15, 2018 by 7 Comments
It will be the moment of truth for the expensive new cholesterol drugs known as PCSK9 inhibitors. Next month we will learn much more about the PCSK9 inhibitor class of cholesterol drugs. A lot of the remaining uncertainty about the efficacy— or lack of efficacy— of these drugs will be resolved when a large cardiovascular…
New Economic Analyses Fail To Make Case For PCSK9 Inhibitors
August 23, 2017 by 1 Comment
(Updated) –PCSK9 inhibitors have ‘the dubious distinction of being the most expensive preventive therapies by far in the history of cardiovascular medicine’ Two new economic analyses conclude that PCSK9 inhibitors are far too expensive to be cost effective. Both studies incorporate data from FOURIER, the first and still the only large cardiovascular outcomes trial with…
Pharma Phantasy: The $30 Billion PCSK9 Inhibitor Market
August 8, 2017 by 2 Comments
–Drug company dreams up a study showing all the benefits and none of the costs or risks of PCSK9 inhibitors. Here’s a pro tip for pushing a drug (or, indeed, any aggressive treatment or screening strategy): focus exclusively on the benefits, even if they are modest at best, and completely ignore side effects and costs….
Forthcoming Expert Guideline May Upgrade PCSK9 Inhibitors
July 21, 2017 by 1 Comment
Results of the FOURIER trial might bolster the embattled cholesterol drugs.Results of the FOURIER trial might bolster the embattled cholesterol drugs.Results of the FOURIER trial might bolster the embattled cholesterol drugs.Results of the FOURIER trial might bolster the embattled cholesterol drugs.Results of the FOURIER trial might bolster the embattled cholesterol drugs.Results of the FOURIER trial…
Ebbinghaus Study May Help Refute Doctor Google
March 18, 2017 by 3 Comments
–No neurocognitive adverse effects linked to very low LDL levels Reaching very low LDL levels with a PCSK9 inhibitor was not associated with any increase in neurocognitive adverse events, according to the largest and most rigorous study to assess the topic to date. Small and preliminary studies have raised concerns over the possibility of the…
Novel Drug Delivers Long Term Cholesterol Reduction
March 17, 2017 by 1 Comment
A novel drug that dramatically lowers LDL cholesterol and needs to be administered only a few times a year has reached a new milestone. Positive results from a phase II study with the drug, now known as inclisiran, were reported at the American College of Cardiology meeting in Washington, DC and published simultaneously in the…
FOURIER Shows New Cholesterol Drugs Work, But Are They Worth It?
March 17, 2017 by 6 Comments
–Doctors and patients now must wrestle with a modestly effective but expensive drug. As it turns out the PCSK9 inhibitor saga ends not with a bang but a whimper. The results of the highly anticipated FOURIER trial show that the drugs work, though not as powerfully as many had hoped and expected. The question now…
27,000 Patient PCSK9 Inhibitor Trial Meets Main Endpoints
February 2, 2017 by 3 Comments
(Updated) –Cardiovascular outcomes finally available for PCSK9 inhibitors. Amgen announced on Thursday afternoon that the FOURIER trial had met both its primary composite endpoint (cardiovascular death, non-fatal MI, non-fatal stroke, hospitalization for unstable angina or coronary revascularization) and the even more rigorous key secondary composite endpoint (cardiovascular death, non-fatal MI or non-fatal stroke). The company…
Genetic Studies Offer Hint Of Clinical Benefits-And Risks- of PCSK9 Inhibitors
November 30, 2016 by 2 Comments
–Both cardiovascular benefits and increase in diabetes seem likely. There is no more eagerly awaited question in cardiovascular medicine than the clinical role of the PCSK9 inhibitors. The first reports from a series of outcomes trials are not due until next year. But two large genetic studies published this week deliver strong indirect evidence that…
More Progress For Novel PCSK9 Drug
November 15, 2016 by Leave a Comment
–The PCSK9 synthesis inhibitor from The Medicines Company is getting close to phase 3 trials. More early information continues to accumulate about a novel cholesterol drug under development by The Medicines Company. The drug, which was initially created by Alnylam Pharmaceuticals, uses RNA interference technology to inhibit the synthesis of PCSK9 in the liver. The main…
Pfizer Ends Development Of Its PCSK9 Inhibitor
November 1, 2016 by 1 Comment
–Immune issues and diminishing efficacy doomed the new drug. Pfizer announced on Tuesday that it was discontinuing development of bococizumab, its cholesterol-lowering PCSK9 inhibitor under development. “The totality of clinical information now available for bococizumab, taken together with the evolving treatment and market landscape for lipid-lowering agents, indicates that bococizumab is not likely to provide…
Repatha Approved for Single Monthly Injection
July 11, 2016 by Leave a Comment
–New hands-free system replaces three sequential shots The FDA has approved a device that can deliver a single monthly injection of evolocumab (Repatha), the cholesterol-lowering PCSK9 inhibitor manufactured by Amgen. Repatha is currently administered by subcutaneous injection in a 140-mg dose every 2 weeks or as a 420 mg monthly dose. Until now, the monthly…
First PCSK9 Outcome Trial Results Pushed Back To 2017
June 8, 2016 by Leave a Comment
Uptake of the new drugs likely to lag without strong outcomes data. Uptake of the new drugs likely to lag without strong outcomes data.Uptake of the new drugs likely to lag without strong outcomes data. (Updated) –Uptake of the new drugs likely to lag without strong outcomes data. There’s probably nothing more eagerly awaited in…
New Cardio Drugs Off To Very Slow Start
April 27, 2016 by Leave a Comment
–Heart failure experts divided over how and when to use Entresto To many long-time observers, the approval last year of two new cholesterol drugs and a heart failure drug appeared to herald a rebirth of the cardiovascular marketplace after a long period of dormancy. But so far in 2016, those new drugs have hardly made…
Not So Rare, FH Is Truly Risky Lipid Disorder
April 3, 2016 by Leave a Comment
–Not all elevated is LDL is equally dangerous CHICAGO — Familial hypercholesterolemia (FH), a condition that has long been overshadowed by more common cardiovascular diagnoses, has now found the spotlight in the big tent as a hot and even controversial topic. The new interest is driven by important advances in understanding the genetics of the…
Court Decision In Patent Battle Threatens PCSK9 Drug
March 23, 2016 by Leave a Comment
—-One analyst thinks the decision might take Praluent off the market. A jury’s decision last week in a patent case might possibly lead to the removal of one of the new PCSK9 inhibitor cholesterol lowering drugs from the US market, according to a Wall Street analyst who has been closely following the case. Amgen, which…
Rat Study Links Neural Tube Defects To Lower PCSK9 Levels
January 3, 2016 by Leave a Comment
(Updated) PCSK9 may play an important role in neural tube development (NTD), a new study in rats suggests. The study, published in Scientific Reports, found that PCSK9 levels were significantly reduced in rat embryos with NTDs. The main focus of the paper was to identify biomarkers that could be used for the prenatal detection of NTDs, since there are now…
First Outcomes Trial Results For PCSK9 Drugs Will Come In 2016
December 21, 2015 by Leave a Comment
In less than a year we may know a whole lot more about the PCSK9 inhibitors than we do now. Since the approval earlier this year of Repatha (evolocumab, Amgen) and Praluent (alirocumab, Regeneron and Sanofi) critics and benefit managers have recommended strict limitations on use of the drugs because of the absence of evidence showing clinical benefit….
Amgen Denies Rumors About PCSK9 Inhibitors (Updated)
December 19, 2015 by 1 Comment
(Updated) Amgen says there is no truth to the rumor that the FDA is investigating post-marketing reports of serious brain infections in patients taking PCSK9 inhibitors. The Amgen statement was in response to this anonymous post on CafePharma: “The FDA informed Amgen and Regeneron today that there have been a number of post marketing reports…
Amgen’s Repatha Beats Out Competitor For Spot On CVS/Caremark Formulary
November 23, 2015 by Leave a Comment
CVS Health announced today that Amgen’s Repatha will be the only PCSK9 inhibitor on the CVS/Caremark formulary. This gives Amgen its first big win among the pharmacy benefits managers against Sanofi’s Praluent in the battle over the market for the powerful but expensive new cholesterol-lowering drugs. Previously Express Scripts had announced that both drugs would be placed…
Amgen Takes The Pledge To Respect Patient Privacy
November 19, 2015 by Leave a Comment
In response to criticism Amgen appears to have greatly tightened its corporate privacy policy regarding patient information. The company has published a “privacy pledge” to US patients” and says that it will soon modify the authorization agreements that required patients participating in the company’s drug copay programs to surrender rights to their personal and health information. The move…
New Definition Of Familial Hypercholesterolemia Could Expand Patient Population For Expensive Cholesterol Drugs
November 5, 2015 by 4 Comments
More people may be diagnosed with familial hypercholesterolemia (FH) using criteria contained in a new scientific statement published by the American Heart Association. The expanded definition could also mean more patients will be eligible to receive expensive cholesterol-lowering drugs, including the new PCSK9 inhibitor drugs, (Repatha from Amgen and Praluent from Sanofi/Regeneron). But the biggest impact might be to increase the…
Amgen Requires Patients in Repatha Copay Program To Surrender Their Privacy
October 22, 2015 by 3 Comments
(This story was updated on October 23 with a statement from Amgen. It was again updated on October 27 with new information about Amgen’s patient privacy policy for a second drug, Enbrel.) (For an important followup to this story please see: Amgen Takes The Pledge To Respect Patient Privacy) The tumult over the new cholesterol…
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