The Food and Drug Administration is bringing together experts next week to advise the agency on whether to approve a version of Genzyme's ( GENZ) Pompe disease drug Myozyme that will be made a new, larger facility. Briefing documents for the Oct. 21 advisory panel, posted Friday, suggest Genzyme may be facing more concerns than previously expected, according to some Wall Street biotech observers. Genzyme does not have enough manufacturing capacity to produce all the Myozyme it requires to treat patients with Pompe disease, a severe and progressively debilitating inherited neuromuscular disorder that affects a very small number of people throughout the world. Efforts to convince the FDA to approve the new version of Myozyme made at a larger manufacturing facility have run into delays. Last April, Genzyme was forced to reduce its 2008 earnings guidance when the FDA asked for more clinical data on the updated Myozyme. The FDA is expected to decide on whether to clear the new version by Nov. 29. Approval is a critical factor for Genzyme's revenue and earnings growth, which is why next week's advisory committee meeting to review the drug is so important. "It's not a slam dunk for Myozyme at this point," said Lazard Capital analyst Matthew Osborne. " The FDA raised a lot of concerns regarding the trial endpoints -- if they were clinically meaningful, the magnitude of response and the insufficient number of patients less than 18 years." To get a large-scale Myozyme manufacturing facility approved,Genzyme provided the FDA with data from a clinical study, dubbed LOTS, that enrolled 90 patients, 81 of whom completed the study.
Stocks soar as the gross domestic product rises at an annualized rate of 3.5% in the third quarter and continuing jobless claims fall. Gregg Greenberg recaps the action in The Real Story video (above).