Published January 11, 2013
A panel of advisers to the U.S. Food and Drug Administration recommended the agency approve an experimental new treatment for diabetes developed by Johnson & Johnson, potentially making it the first drug of its type to be approved in the United States.
The FDA's Endocrinologic and Metabolic Drug Advisory Committee voted 10-5 on Thursday to recommend the agency approve the drug, canagliflozin, for Type 2 diabetes, saying that it proved effective at lowering blood sugar in patients with diabetes, though some panelists had lingering concerns about its potential to cause cardiovascular problems and recommended longer term follow-up.
Canagliflozin, which will be sold under the brand name Invokana, is a member of a new class of diabetes drugs known as sodium-glucose co-transporter-2 (SGLT2) inhibitors which lower blood sugar by blocking reabsorbtion of glucose by the kidney and increasing the excretion of glucose in urine.
In its discussion, the panel weighed the relative risks and benefits of canagliflozin, especially in relation to any potential it might have to increase the risk of heart attack or stroke.
A clinical trial of patients at especially high risk of cardiovascular disease showed that within the first 30 days, 13 patients taking canagliflozin suffered a major cardiovascular event compared with just one patient taking a placebo. After that the imbalance was reversed. The drug also caused a slight increase in unhealthy LDL cholesterol.
The majority of panelists felt the overall risk benefit profile was acceptable but that longer-term data will be needed to fully assess the impact on patients of the higher LDL levels. They were unable to determine conclusively that the imbalance in cardiovascular events seen in the first 30 days was a statistical anomaly.
Diabetes is a condition that affects the body's ability to metabolize glucose and is often caused by obesity. Left untreated, the disease can cause nerve disease leading to amputation, as well as kidney disease and blindness. It affects roughly 26 million people in the United States.
The panel also weighed the relative benefit of the drug for patients with impaired kidney function -- a common feature of patients with diabetes. They concluded that since the drug is less effective in patients whose kidney function is damaged, the risks may well outweigh the benefits in those patients.
Jeff Jonas, an analyst with Gabelli & Co, who estimates the drug will generate at least a billion dollars in annual sales for J&J, said he believes the FDA will approve the drug.
"It clearly works, and the side effects were not a major issue. If a patient has impaired kidneys, I think the FDA will say no, don't use it."
Damien Conover, an analyst at Morningstar, believes the drug could generate peak annual sales of more than $2 billion.
The vote in favor of canagliflozin follows the agency's rejection last January of a similar drug made by Bristol-Myers Squibb Co and AstraZeneca Plc. That drug was subsequently approved in Europe, however, under the brand name Forxiga. European regulators concluded that concerns cited by the FDA about a potential increased risk of cancer or liver injury were addressed by warnings in the drug's product label.
A recent report by market research firm Decision Resources estimated that the market for Type 2 diabetes drugs will nearly double over the next decade, increasing from $26 billion in 2011 to nearly $50 billion in 2021 in the United States, Japan and the main markets of Europe.
The FDA is set to rule on whether to approve the drug by March 29th. The agency is not required to follow the advice of its advisory panel but typically does so.