At last Genzyme Corp has received some good news after it emerged that the US Food and Drug Administration has given the green light to the troubled company’s Pompe disease treatment Lumizyme.
The regulator has approved Lumizyme (alglucosidase alfa), which is produced in 4,000-litre bioreactors at its facility in Geel, Belgium, not at the Allston Landing, Boston plant which was temporarily shut down after a bioreactor was contaminated with a virus. Then, in November, the FDA warned of the potential for foreign particle contamination in a number of Genzyme therapies, including another Pompe disease therapy, Myozyme (also alglucosidase alfa).
Genzyme had originally filed to manufacture the drug in Allston Landing but following the manufacturing woes at the site, the FDA rejected that idea. Therefore the company, which has spent more than 10 years and $1 billion developing Lumizyme, needed to come up with another approach, including the plan to make it in Belgium.
The FDA approval now means Genzyme will be able to sell Lumizyme to about 200 adults in the USA who have been receiving it for free under a charitable programme and to another 1,000 people who also have Pompe disease but are not yet being treated; the cost of the treatment is about $300,000 per year.
Genzyme chief executive Henri Termeer noted that the approval came ahead of the scheduled Prescription Drug User Fee Act date of June 17. The company noted that patients must be enrolled in a risk evaluation and mitigation programme and the drug will carry a boxed warning reflecting the risk of life-threatening anaphylactic reactions, severe allergic and immune-mediated reactions.
The news has gone down well with analysts, coming as it does just a day after Genzyme said it would give the US government $175 million from the sale of products that were made at the Boston facility as part of a consent decree agreed with FDA. Christopher Raymond at Robert W Baird issued a research note saying that the approval gives the firm a “much-needed credibility boost after a steady stream of bad news”.
