FDA fast-tracks "breakthrough" heart failure drug

Entresto pill for heart failure patients with reduced ejection fraction, which received F.D.A fast-track approval July 7, 2015.

Novartis

Last Updated Jul 9, 2015 2:16 PM EDT

A new drug for heart failure, Entresto, received a fast-track approval from the FDA on Tuesday, six weeks earlier than expected.

The new drug, previously known as LCZ696, is the first to show a higher reduction in deaths from heart failure and trips to the hospital versus the existing standard. Testing against the most common drug, enalapril, Entresto showed about a 20 percent reduction in those categories.

"For those of us who have been practicing in heart failure, we've been waiting for a day like this for 15 years, which was the last time we had a major breakthrough in the treatment of systolic heart failure," Dr. Sean Pinney, Director of the Advanced Heart Failure and Transplantation Program at The Mount Sinai Hospital told CBS News. "It reduces heart failure hospitalizations and it helps people with heart failure live longer--and it's powerful."

Fast-track approval is reserved for drugs the FDA believes will fill an unmet medical need to treat serious or life-threatening conditions. The review process for the drug was expedited because it was already viewed as filling a gap.

The study they used to review and give expedited approval to Entresto was the largest ever trial of a heart failure treatment. Approximately 8,400 people were divided into groups and, along with their other medications, were either given this new drug or the standard enalapril, an angiotensin converting enzyme (ACE) inhibitor.

Entresto users had a 21.8 percent rate of deaths or hospitalization within 27 months, while enalapril users had a 26.5 percent rate. Entresto is different from ACE inhibitors because it's a dual-component drug that combines the company's blood pressure drug, Diovan (valsartan), with the drug sacubitril, which inhibits an enzyme present in heart failure, called neprilysin.

"Entresto is expected to change the management of patients with [heart failure and reduced ejection fraction] for years to come," principal investigator of the study, Dr. Milton Packer, Professor and Chair for the Department of Clinical Sciences at University of Texas Southwestern Medical Center said in a statement. He described the results as showing a, "very meaningful survival advantage."

Typical side effects of Entresto are like those of other heart failure medications: low blood pressure, high potassium levels and poor kidney function.

More serious reactions that concern doctors in treating heart failure patients are specific to Black patients and people who cannot tolerate the decrease in blood pressure, which can cause dizziness and falls.

"The side effects are real and I have some lingering concerns," said Pinney. "I'm going to be cautious about using it in African Americans and look to see whether or not they're more susceptible to developing angioedema, which is the swelling of the tongue and the lips."

"As we begin to treat more real world patients with this drug, I suspect there are going to be drops in blood pressure that we're going to have to be cautious about," he added.

According to the CDC, heart failure is associated with more than one in nine deaths and costs about $32 billion dollars per year. Once diagnosed with heart failure, patients generally live about another five years, on average. They are usually prescribed drugs to take for the remainder of their lives.

An estimated 2.2 million patients could be treated with Entresto. It is approved for people who have a combination of heart failure -- when the heart is too weak or stiff to pump blood at the normal capacity -- combined with reduced ejection fraction -- a measure of how well the heart pumps with each beat. The manufacturer, Novartis, wants to gain approval to use Entresto for even more of the estimated 5.1 million people living with heart failure in the U.S, according to the CDC, through upcoming studies.

But the increased survival rates don't come without a cost. The drug will be expensive, particularly for those whose insurance may not cover it completely. Novartis says that it will price Entresto at about $12.50 per day, but may adjust the price depending on how effective it is at keeping people out of the hospital when it's used by a broad range of people. They may also offer incentives for lower-income patients.

"The benefits in preventing hospitalization and extending survival will be weighed against the price of the drug," said Pinney.

Novartis anticipates the drug will have a large market and represent big profits for the company, potentially more than $5 billion worldwide.


This story was updated to correct an error in the headline. Entresto was not the first new heart failure drug approved in the past decade;Corlanor received FDA approval earlier this year.