FDA Says It Won’t Approve Amylin’s Diabetes Treatment Without More Data

Amylin Pharmaceuticals Inc. suffered a setback Wednesday when the Food and Drug Administration said it wouldn’t approve the company’s experimental diabetes drug without additional information.

The San Diego firm has been developing the drug for 16 years.

Called Symlin, it is a synthetic version of a natural hormone that works with insulin to regulate the flow of sugar from food into the bloodstream.

Amylin indicated that the FDA said it was concerned about Symlin’s effect on hypoglycemia, or low blood sugar, which can lead to nausea, confusion and brain injury and that the agency asked for more information on its safety and benefits.

In 2001, a panel of experts recommended against FDA approval of Symlin, in part because of similar worries. After the rejection, Amylin completed a 296-patient trial this year in people with Type 1 diabetes and submitted results to the FDA.

Now the FDA wants data from Amylin’s clinical trials that identify either the type of patients who don’t have increased risk of “significant hypoglycemia,” or the patients for whom Symlin would justify the risk.

Amylin Chief Operating Officer Daniel M. Bradbury said the company believed it has that information but couldn’t be more specific. “It is difficult to interpret what the agency might require,” he said. The company hopes to meet with the agency soon, he said.

Amylin’s stock fell 20 cents to $23.43 on Nasdaq before the company announced the FDA news. Shares have climbed 45% this year as investors anticipated the injectable diabetes drug would be approved for sale.

Symlin would have been the first drug approved for Type 1 diabetes since insulin hit the market nearly 80 years ago.

The product, Amylin’s first, was expected to reach the market by early next year and have 2004 sales of $60 million, said Russ Gilbertson of Roth Capital Partners. He expected sales to reach $300 million in 2007.

John McCamant of the Medical Technology Stock Letter in Berkeley said he believed Symlin would eventually be approved. Judging from Amylin’s news release, he said, the FDA is asking for data it should have sought before giving the go-ahead for the latest clinical trial.

“The FDA is supposed to help a company design a clinical trial to get the data it needs. It sounds to me like the FDA isn’t doing its job,” McCamant said.

Patients with Type 1 diabetes, the kind that often strikes in childhood or adolescence, are dependent on insulin shots to control spikes in blood sugar, which can lead to coma and death.

Regular insulin shots can control the disease, but high blood sugar can take its toll on patients later in life who may suffer blindness, kidney failure and heart disease. Symlin was developed as an additional tool to control blood sugar. In early human tests of Symlin, the drug combination lowered blood sugar too much in some.

An Amylin spokesman said the its most recent clinical trial showed that fewer patients had hypoglycemia if they adjusted their insulin dose.

Symlin “is a major breakthrough in the treatment of Type 1 diabetes,” said diabetes expert Dr. Rubina Heptulla of Baylor College of Medicine in Houston. “Now we have nothing to offer patients but more insulin.”

Diabetes affects 18 million adults in the U.S., twice the number 10 years ago.

Amylin’s quest to develop Symlin has suffered a series of financial and scientific setbacks. It came within weeks of closing its doors in 1998 after Johnson & Johnson pulled out of a deal to co-develop the drug. The company has spent $300 million to develop Symlin.

It is not Amylin’s only experimental drug for diabetes. Eli Lilly & Co. will invest as much as $325 million to co-develop with Amylin a drug for Type 2 diabetes that analysts believe could be a $1-billion product.