Biotech Values

[DewDiligence]

FDA panel backs the “new” Myozyme by 16-1 vote,
asking for additional study on postmarketing basis.

http://www.reuters.com/article/marketsNews/idINN2132472020081021

›Panel Backs Mass-Produced Myozyme

Tue Oct 21, 2008 7:12pm EDT
By Susan Heavey

SILVER SPRING, Md., Oct 21 (Reuters) - A mass-produced version of Genzyme Corp's (GENZ) drug to treat the fatal genetic disorder Pompe disease should be approved as long as further studies are required, a U.S. advisory panel said on Tuesday.

Data for the intravenous drug, already sold on the market as Myozyme but produced at a smaller facility, showed the drug worked in patients who developed Pompe disease after infancy, the U.S. Food and Drug Administration's panel of outside advisers said in a 16-1 vote.

"This would be a nice compromise to be able to have the drug approved but still make sure that there are postmarketing studies to ensure there's safety and efficacy," panel chairman Dr. Kenneth Burman of the Washington Hospital Center told reporters after the meeting.

Genzyme is seeking FDA approval to sell its new version, made with a 2,000-liter capacity bioreactor, so that it can increase availability. Its version made with a 160-liter reactor was approved in 2006.

But because the product is a biological compound made with complex proteins, the FDA earlier ruled the mass-produced version must be considered separately.

Genzyme wants to sell 2000-L Myozyme, also known as alglucosidase alfa, to treat Pompe disease patients who develop the rare condition after age two.

The debilitating enzyme disorder causes patients to progressively lose use of their muscles. The lack of the alpha-glucosidase (GAA) enzyme causes a build up of stored blood sugars that weaken the heart, make it hard to breathe, and ultimately cause death.

The condition affects between 5,000 and 10,000 people worldwide, according to Genzyme.

At the meeting, FDA staffers questioned the company's clinical trial design and said there was not enough data to approve it for patients younger than 18 years old.

Genzyme officials, however, said if the FDA imposed age restrictions, the company could not make enough 160-L Myozyme to supply all the children under age 18 who need it. The company's 2000-L Myozyme is already approved in 43 countries, they said.

Patients at the meeting urged the FDA panel to support 2000-L Myozyme, saying the bi-weekly enzyme replacement therapy helped them breathe and stay mobile enough to keep their jobs and interact with their families.

"I'm weak ... my balance is shaky," said Brian White, a patient from Virginia. Taking Myozyme, he said, has "basically allowed me to continue my life the way it is today."

FDA's advisers, compelled in part by such testimony, agreed even though they said the data were not overwhelmingly strong. Additional studies should take a closer look at how the drug works and its risks, which include life-threatening reactions such as shock.

"Taken altogether... these data suggested that the drug did have benefits, and if the drug had benefits, we didn't want to exclude it from patients who might benefit," Burman said.

But Dr. John Teerlink of the University of California, voted against the 2000-L product, saying there was no reason to support approval without conclusive evidence that it helped patients live longer or feel better. "I think we're being given a false choice… The option is for the company to make more 160-liter generators if it needs to," he said.

The advisory panel's votes straddled the close of regular trading on Nasdaq. After gaining 2.3 percent to close at $68.32 in the regular session, the shares rose 2.5 percent to $70 in extended trading.

Analysts have had mixed opinions on whether the FDA's concerns will delay approval.

The agency's decision is due by Nov. 29, but it has come under recent scrutiny for missing its deadlines. Sandra Kweder, deputy director of the FDA's Office of New Drugs, said the agency strives to meet such goals, but it was not clear if it would meet the one for Myozyme.‹


<font size=3><font color=red> “The efficient-market hypothesis may be
the foremost piece of B.S. ever promulgated
in any area of human knowledge!”