A Food and Drug Administration advisory panel voted largely in
favor on Tuesday regarding the company's bid to make its Pompe disease drug Myozyme at a new, larger facility.
Genzyme shares were up $1.19, or 1.7%, to $69.51 in post-market trading.
The FDA isn't bound by the recommendations of its panels, but it often does follow the sentiment of its advisers. The agency is set to decide Nov. 29 on whether to approve the larger-scale version of Myozyme for late-onset patients. The population encompasses juvenile and adult patients with Pompe disease, a rare but severe and progressively debilitating inherited neuromuscular disorder.
Late last week,
and its investors were surprised by concerns raised in FDA briefing documents regarding the effectiveness, safety, and age representation in the trial that was the basis for the company's application.
On Tuesday, however, the panel voted 15-1 that the LOTS study established the efficacy of Myozyme produced at the larger scale and 16-0 (with one abstention) that the newer product should not be restricted to the adult-onset population, meaning patients who were diagnosed and had symptoms begin after the age of 18.
Notably, the advisory committee voted that post-marketing studies should be required to continue assessing the updated Myozyme's effectiveness and safety.
Myozyme produced at a smaller scale is currently approved in the U.S., but Genzyme doesn't have enough manufacturing capacity to produce all the drug it needs to treat patients with Pompe disease. Thus, it has been seeking clearance to produce the drug in a new manufacturing facility designed to make Myozyme at a larger scale.
Last April, U.S. regulators decided that Myozyme made in a 2,000-liter plant was different than the one currently made at the 160-liter plant, and as a result, said they would require a separate biologics license application supported by a clinical trial.
Genzyme is currently providing the drug out of the 2,000-liter facility for free to about 160 patients through a compassionate use program, which is costing the company roughly $140,000 per patient each year, according to Deutsche Bank analyst Mark Schoenebaum.
Upon approval, the drug produced at the larger-scale plant will be commercially available to the patients enrolled in the program.
The number of patients in the program, however, has already brought the new larger-scale facility to full capacity, according to Schoenebaum. Thus, he says that even with approval of the facility, patients who are not receiving the drug through the program will have to wait for approval of a 4,000-liter plant in Europe, which is expected in the first half of 2009.
Genzyme is set to report its third-quarter earnings Wednesday morning, followed by
after the market closes and