September 3, 2014

Novartis Announces "Game-Changing" Drug to Treat Heart Failure

The biggest medical news this past week – some might say in the past decade – was the announcement that a new drug has been shown to significantly improve the chances for the estimated 26 million people in the world living with heart failure.

This announcement underscored two important realities for me, one uplifting and the other sobering:
  1. Important medical breakthroughs – in any arena -- are indeed still possible.
  2. Unfortunately, those breakthroughs are possible only when Big Pharma stands to cash in.
In this most recent widely-heralded case, Swiss giant Novartis’s new still-unnamed drug (LCZ696) – when compared to the best treatment now available (enalapril) -- showed these impressive results:
  • reduced the risk of death from cardiovascular causes by 20%
  • reduced heart failure hospitalizations by 21%
  • reduced the risk of all-cause mortality by 16%
Novartis announced the study results on August 30, 2014 at the European Society of Cardiology Congress in Barcelona, Spain. The findings were published simultaneously in the New England Journal of Medicine.

Reaction from the medical community was swift and very enthusiastic.

"This is a new day" for patients, said Dr. Clyde Yancy, cardiology chief at Northwestern University in Chicago and a former American Heart Association president. He played no role in the study. "It's been at least a decade since we've had a breakthrough of this magnitude," he added.

“I think that when physicians see these data, they will find it compelling, and what we will see is a paradigm shift,” said Dr. Milton Packer, a professor of clinical sciences at the University of Texas Southwestern Medical Center in Dallas and one of the two principal investigators in the study.

Mariell Jessup -- a heart failure expert at the University of Pennsylvania -- wrote in the NEJM’s glowing editorial that LCZ969 “may well represent a new threshold of hope for patients with heart failure."

What Exactly is Heart Failure?
Heart failure is a debilitating, potentially life-threatening disease. It hospitalizes more older people than any other condition, and is a leading cause of death.

The National Institutes of Health (NIH) describes it this way:
Heart failure is a condition in which the heart can't pump enough blood to meet the body's needs. In some cases, the heart can't fill with enough blood. In other cases, the heart can't pump blood to the rest of the body with enough force. Some people have both problems. 
The term "heart failure" doesn't mean that your heart has stopped or is about to stop working. However, heart failure is a serious condition that requires medical care. Heart failure is a very common condition. About 5.1 million people in the United States have heart failure.

That definition explains why this particular Novartis announcement was such a big deal. Heart disease is a major health issue, and LCZ696 apparently shows great promise to treat it.

The new drug works using a twice-a-day pill combination of two medicines. One blocks the effects of substances that harm the heart while also preserving substances that help protect it. One of the medicines also dilates blood vessels and allows the heart to pump more effectively.

A Big Breakthrough after a Big Trial
This study was a randomized, double-blind, phase III trial -- the largest heart failure experiment ever conducted – evaluating the efficacy and safety of LCZ969 versus enalapril, the most widely used ACE inhibitor heart failure treatment (sold in generic form and also as Vasotec).

The test followed 8,442 patients in 47 countries for almost five years. Study participants were already taking three to five medications for heart failure. In March 2014, independent monitors ended the study earlier than planned when the data clearly showed that the new drug worked better than the old one.

LCZ969 also seemed safe, an important consideration since safety concerns had torpedoed several other promising heart failure treatments over the past ten years. In LCZ969’s case, there was increased evidence of hypotension (low blood pressure) and non-serious swelling under the skin.

However, the new Novartis drug avoided the key troubling side-effects that accompanied enalapril use: kidney problems, excess potassium in the blood, and cough.

Ka-Ching: Hear the Cash Registers Ring
Here’s the part I expected: the study was paid for, designed, and partly run by Novartis, the Swiss pharmaceutical behemoth.

Touted Novartis Pharmaceuticals chief David Epstein, “It isn't often that you get a drug that saves lives, reduces costs and makes people feel better."

The drug giant plans to complete its application for LCZ969 with the Food and Drug Administration by the end of the year, and the FDA has already granted the drug a priority review.

Analysts at Deutsche Bank estimate that LCZ969 could eventually generate up to $10 billion a year – a major blockbuster. Drug launches over the past few decades have been aimed at smaller, more targeted patient populations.

Analysts at Sanford C. Bernstein & Company in New York predict the new drug could launch at about $7 a day ($2,555 a year) in the United States and $4 in Europe, where drug prices are lower. They noted that other companies had very few competing products in development. 

Novartis’s Epstein said LCZ696 could reach the market by the third quarter of 2015 – a boon, he explained, to the 1 in 5 adults who will get heart failure in their lifetimes… 50% of whom will not live more than five years. 

Though he wouldn’t comment on the drug’s pricetag, he made a strong medical and economic case for its use:
This disease is every bit as bad as metastatic cancer. At a time when the world is focused on the economics of health care, when you see a drug that reduces hospitalizations so markedly, this is a drug that’s not just about helping people live longer, it could help address some of the economic issues.
Maybe so. One thing IS sure: it could make Epstein’s company more money than he ever imagined possible. 

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