Good news at last for Genzyme, which gets FDA approval for Lumizyme earlier than anticipated

Troubled US biotechnology firm Genzyme has at last received some good news, with the Food and Drug Administration granting marketing approval for Lumizyme (alglucosidase alfa), the first treatment approved in the USA specifically to treat patients with late-onset Pompe disease, in a decision that came ahead of schedule.

Lumizyme is the US trade name for alglucosidase alfa, which is sold in Europe as Myozyme and is in restricted supply do in part to chronic problems at one of the product's key US manufacturing facilities. Lumizyme is to be produced at the 4,000 liter bioreactor scale at its manufacturing facility in Geel, Belgium. Shares of Genzyme shot up more than 6% to $51.40 following the news, in a sharply downturned broader market, where the Dow Jones fell below the psychologically important 10,000 mark, pleasing investors who had feared the FDA would delay a decision until the company had rectified its production problems, relating to which Genzyme this week agreed to pay back 'illegal profits' of $175 million and made other commitments (The Pharma Letter May 25).

'This is an important day for the Pompe community, especially for those patients with late-onset Pompe disease in the United States who are awaiting treatment for this devastating disease,' said Genzyme chairman and chief executive Henri Termeer. 'We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date,' he added.