We’ve heard a few presentations from Genzyme about Pompe disease, a rare respiratory and musculatory disorder afflicting one in 40 to 300,000 people. (It’s an “orphan” condition, and Genzyme’s intravenous drug, Myozyme, was approved for Pompe disease in infants in April, 2006.)

But we had not been previously aware that the company was working on additional indications for its product using adaptive clinical trial designs. Here’s an excerpt from a recent Genzyme presentation at an investor meeting:

“Last year, the company obtained approval for Myozyme (alglucosidase alfa), the first product ever developed for Pompe disease, a progressive, debilitating and often fatal neuromuscular disorder. Myozyme is off to a strong start. It was launched in Europe and the United States during the second quarter of 2006, and more than 550 patients in approximately 35 countries are currently on therapy.

Sales grew significantly in the fourth quarter, increasing to $30 million. This year, Genzyme expects to launch Myozyme in Japan, Brazil and a number of additional markets. Myozyme recently received the prestigious Panorama del Medicamento award as the most innovative drug of 2006 in Spain, as deemed by the National Pharmaceutical Council, the highest professional body of Spanish pharmacists. In September, Myozyme earned the 2006 UK Prix Galien Gold Medal, presented bi-annually to new products judged to be the most innovative.

Results from the pivotal clinical trial of Myozyme were published last month in Neurology. Results from the ongoing study of Myozyme involving patients with late-onset Pompe disease are expected later this year and will be submitted to regulatory authorities in 2008. The 90-patient trial is intended to provide further support for Myozyme’s use. Using an innovative adaptive design procedure, the duration of this trial has been extended for six months and will now end this fall when all patients will have completed 18 months of treatment.”

On its face, unfortunately, that could be cryptic to those outside the industry. It might have been written a bit better. Read literally, Genzyme’s statement appears to suggest the “adaptive” approach will lengthen, not shorten, the cumulative scientific odyssey to the end of the trial. We’re guessing that is not the case, but what do we know?

All the same, Genzyme’s statement is welcome. It’s an unusually rapid, public acknowlegment of an innovative strategy that could inspire others to explore the same territory. It will be interesting to see if the company elects to delineate how much time or money were saved using the adaptive approach.

It would also be nice to see whether Genzyme might be persuaded to specify all of the tools (and partners) it relied upon in Myozyme’s development at conferences over the coming months. There must be something for the rest of the industry to learn from this.

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