Friday, February 27, 2009

EU approves Genzyme’s Pompe disease drug

By Marc Songini

Genzyme Corp. has received European approval for manufacturing Myozyme, an anti-Pompe disease drug.

This week, Genzyme (Nasdaq: GENZ) announced that the European Commission gave the thumbs-up for the production of Myozyme at the 4,000-liter bioreactor scale at the company’s Geel, Belgium-based factory. The firm said that with this approval, it can supply Myozyme to all Pompe disease patients in Europe. During January and February, it was necessary for adults to adjust their drug-infusion schedules to ensure there was a product supply for infants and children. The approval now means adult patients can resume regular infusion schedules and new patients can start treatment.

In turn, sales are expected to jump in the second quarter and continue to increase for the second half of the year. Genzyme anticipates Myozyme sales for the first quarter of 2009 to be consistent with those of the fourth-quarter 2008.

Cambridge-based Genzyme claimed that Myozyme (alglucosidase alfa) is the only approved treatment for Pompe disease, an inherited disease that’s progressively debilitating and frequently fatal. All patients usually face progressive muscle weakness and breathing difficulty, but the rate of disease often depends on such things as the age of onset. If symptoms appear during the few months after birth, affected babies frequently suffer an enlarged heart and die in the first year. For children, adolescents and adult patients, there is a progressive debilitation and premature mortality through respiratory failure. Patients also often require mechanical ventilation and wheelchairs. The disease affects fewer than 10,000 people worldwide.

Genzyme has more than 11,000 employees worldwide and had 2008 revenues of $4.6 billion. This month, it also announced the U.S Food and Drug Administration had granted it approval for its viscosupplement Synvisc-One, for the relief of pain caused by osteoarthritis of the knee.