Amylin Pharmaceutical Inc.'s approvable letter for its lead drug candidate Symlin for Type I and Type II diabetes marks a major turnaround for the biotechnology firm.
In July, a Food and Drug Administration panel voted 8-1 against using Symlin for Type I patients and 6-3 against its use for Type II diabetes.
The panel also voted 8-1 that the trials proved efficacy, but in another 8-1 vote they said the drug appeared not to be safe.
Typically the FDA follows the ruling of its panel, but not this time.
"I thought it was a surprising vote," said Fariba Ghodsian, an analyst with Cruttenden Roth Inc. in Los Angeles. "The FDA mostly follows the recommendations of the panel."
She added the FDA is usually even more conservative than the panel.
The support shown by some panel members, however, spoke for the drug.
The FDA said it wants to see more data before it will approve Symlin for marketing. Amylin officials could not say when they'll meet with regulators to discuss details of the trial.
Mark Foletta, Amylin's CFO, said the firm hopes to sit down with FDA officials soon and discuss "what kind of clinical work is necessary to address their concerns."
Foletta did not want to speculate on a time frame.
Ghodsian said the FDA reviewers had a major concern with severe hypoglycemia which is when blood-sugar levels become dangerously low. Hypoglycemia is often associated with unconsciousness and "major trauma, motor vehicle accidents and death."
The risk of hypoglycemia was especially high in patients during the first four weeks of treatment. Consequently, the FDA is likely to demand a trial focusing on the first month of treatment to see if adjustments in dosing, where patients are gradually exposed to the drug, can prevent hypoglycemia, she added.
But even if Symlin is approved, getting doctors to adopt the drug may be challenging.
Symlin, which is an analogue to the human hormone amylin, needs to be injected three times a day in conjunction with insulin.
"Since the drug would be used in addition to insulin, the question is whether a similar benefit could be achieved by just giving insulin more frequently," Ghodsian said.
Ghodsian said the drug could generate about $260 million worldwide at peak sales and would likely be on the market in 2003.
Foletta said Amylin plans to market Symlin itself with about 150 sales reps that would pitch the drug to a small group of doctors.
According to market research, about 21,000 doctors write 50 percent of all insulin prescriptions in the United States, Foletta said.
He said the firm hopes to gain acceptance by educating doctors on "the role of (the hormone) amylin in normal physiology and deficiency in diabetes."
In addition to the good news on Symlin, the FDA informed Amylin that it approved an application to take its second drug candidate AC2993 into Phase III trials, the last of three phases required by the FDA to approve a drug.
"We work with the same division of the FDA. (That's a) signal we got that group's attention," Foletta said.
He added while it's too soon to celebrate, the prospect of getting two diabetes drugs on the market is exciting.
- - -Bankruptcy:
Akkadix, a La Jolla-based agricultural biotechnology firm, has filed for Chapter 7 bankruptcy and laid off its work force.
In its filing Sept. 24, the privately held company said it was unable to attract investors. It also blamed the events of Sept. 11, which threw the marketplace into disorder.
The company was formed in 1999 by the merger of Global Agro and Xyris Corp.
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