Genzyme made it one step closer to marketing its Pompe disease therapy, Myozyme (alglucosidase alfa), last week when it submitted its regulatory dossier to the US Food and Drug Administration seeking the green light of approval. If cleared, Myozyme would become the first treatment for patients with this potentially fatal muscle condition caused by an inherited enzyme deficiency.
The dossier includes data from several clinical trials, including the latest to demonstrate a positive outcome involving 18 patients with infantile-onset Pompe disease who received Myozyme from six months of age. At 18 months, 83% of patients were alive and free of invasive ventilator support, compared with just 2% who were alive in the historical cohort.
Genzyme is likely to receive priority review for Myozyme, meaning that it could hear a decision on approvability within the forthcoming six months. Said Henri Termeer, chairman and chief executive: “Myozyme could be available in the USA and Europe by the early part of next year and in other parts of the world relatively soon after.” The company plans to file these new data with European regulatory authorities as part of a dossier submitted in December last year, and will seek a nod from Japan later on in 2005.