Repatha's clinical trial involved over 27,000 patients and cost over $1 billion. ['The City' from Herry Lawford]
Repatha’s clinical trial involved over 27,000 patients and cost over $1 billion. [‘The City’ from Herry Lawford]

We just learned a lot more about a drug called Repatha, one of the newest weapons in the war against heart disease.  Heart-related diseases are the leading cause of death worldwide, accounting for over 30% of global deaths.  One of the major physiological warning signs of heart disease is “bad” cholesterol (LDL), which can clog arteries and increase the risk of heart attack and stroke.  Repatha, developed by Amgen, reduces LDL by blocking the activity of a protein known to prevent LDL from being removed from the bloodstream.

The science looks good on paper, but how well does it work in humans?  A recent clinical trial showed that Repatha patients saw massive declines in LDL levels and experienced fewer heart attacks and strokes.  Unfortunately, and surprisingly, these gains did not translate into longer lives.

So what happened?  Despite advances in genetics, chemistry, and molecular biology, it’s still exceedingly difficult to predict how changes on the molecular scale will affect someone’s long-term health.  For example, a candidate drug called torcetrapib, which lowered LDL and raised “good” cholesterol, didn’t decrease mortality either.  In fact, torcetrapib actually increased its patients’ risk of death.  Collectively, these results suggest that the relationship between cholesterol and heart disease-related mortality may be more complicated than originally thought.

While Repatha shows clear benefits, it may have raised more questions about heart disease than it answered.

Acknowledgments: Many thanks to Michael Macarthur, a Ph.D. student in the Biological Sciences in Public Health program at Harvard University, for providing his expertise and commentary on the topic.

Managing Correspondent: Christopher Gerry

Original Research: Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease – The New England Journal of Medicine

Media Coverage: Cholesterol-Slashing Drug Can Protect High-Risk Heart Patients, Study Finds The New York Times

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