This Diabetes Drug Saves Lives. You Can Thank The FDA

This evening, researchers are announcing that Victoza, a diabetes drug sold by Danish drug giant Novo Nordisk , prevents heart attacks, strokes and cardiovascular deaths.

It is only the second diabetes drug ever to do so. The first, Jardiance, a pill sold by Eli Lilly and Boehringer Ingelheim , presented its positive results just last year. Researchers say that the new results could change the way that doctors treat diabetes, shifting the treatments doctors reach for after metformin, the tried-and-true first-line drug, which is generic.

“There’s a building momentum that maybe we do need to rethink the way diabetes is cared for in America,” says John Buse, the University of North Carolina, Chapel Hill researcher who led the study, which was funded by Novo Nordisk.

And doctors and Novo Nordisk itself give credit to the new diabetes data to a surprising source: Tougher regulations for diabetes drugs from the Food and Drug Administration, which many in industry had previously decried, saying it was keeping new drugs from the market and hurting patients.

“I can almost guarantee you that these trials would not have been done if it had not been for the FDA regulations,” says Buse, who has been a consultant to many companies for years. “Before the guidance I was constantly pushing on companies to do these trials.”

That fact–that companies and patients are likely to benefit from the FDA’s toughness–goes against one of the common narratives in the drug industry and among the FDA’s critics: that high regulations slow patients’ access. In some cases, it's clear, they also create a bar for industry to leap over, and deliver billions of dollars in spoils to companies that actually manage to help patients, not just blood test results.

The Victoza result is exactly the kind of marketing claim that makes a drug company salivate: Novo Nordisk can now tell patients and their insurers that the alternative to its drug is an earlier death.

In the study, presented this evening at the annual meeting of the American Diabetes Association and published in the New England Journal of Medicine, 9,340 patients were randomly assigned to receive either Victoza or placebo for a median of 3.8 years. For those on Victoza, 13% had a heart attack, stroke or death, compared to 14.9% on placebo, a 13% decrease in risk. Reductions in cardiovascular death (22%) and death from any cause (15%) were also statistically significant. A supposed side effect of the drug, pancreatitis, did not show up at all, and patients on Victoza lost 2.3 kilograms (about 5 pounds) more than those on placebo.

The results are less puzzling than those for Lilly and Boehringer’s Jardiance, which seemed to have a benefit not from reducing artery disease, but from its effect on blood pressure or some other mechanism. That drug, though, had a very clear impact on death.

Mads Krogsgaard Thomsen, Novo Nordisk’s executive vice president for research and development, says he believes the results will help drugs like Victoza that work by mimicking the glucagon-like peptide one (GLP-1), a substance the body uses as signal to help control blood sugar. Eli Lilly’s Trulicity and GlaxoSmithKline’s Tanzeum are other drugs in this class. He also thinks it will help Victoza gain market share. A follow-up drug from Novo Nordisk, semaglutide, which needs to be given less often than Victoza, has already prevented heart attacks, strokes and deaths in a 3,000-patient trial; full results are forthcoming.

“To be honest, I would give FDA the credit they deserve,” Thomsen says. "Because there were many, many skeptical people in the industry that said it was overkill for the industry to be asked to do major cardiovascular studies to prove the safety of their medicines. Had it not been for the FDA’s demand for these trials, we would not know that we have two lifesaving drugs.”

The tests of the new diabetes drugs were birthed by one of the biggest controversies to hit the FDA: the accusation that the best-selling diabetes pill, GlaxoSmithKline’s Avandia, caused heart attacks. Steven Nissen, who launched the controversy with an analysis in the New England Journal of Medicine, suggested at an FDA meeting that what were needed were large studies of whether new diabetes drugs prevented heart attacks. These studies, which were attacked as a barrier to new products, are what are now delivering results.

“It is a triumph for evidence-based medicine,” Nissen says of the two trials. (He worked on the Victoza study.) “It is incredibly important for patients. It will stimulate industry to work even harder to try and find therapies that will affect cardiovascular mortality. It’s an amazing turnaround for a field that was stagnating.”

It’s not certain yet that the drugs will see a sales boost as a result of the data. Last year, Jardiance had sales of only $114 million; analysts at Bernstein Research expect that number to rise to $2 billion by 2020. Victoza had annual sales of $2.7 billion last year, which Bernstein expects to increase to $3.9 billion by 2020. The biggest victim of these increases: Merck’s Januvia, a popular pill with no proven benefit on heart attacks and strokes and $6 billion in sales.

Doctors will still tend to use metformin first in part because of price. Jardiance costs more than $4,000 a year, an Victoza can cost $6,000 or more, depending on dose.