FDA Grants Priority Review for Genzyme’s Cerdelga
US regulatory body, the Food and Drug Administration (FDA), have announced that they will be undertaking an accelerated a review of Sanofi’s Genzyme unit’s Gaucher disease drug, Cerdelga (eliglustat), having granted a six-month priority review for the application.
Gaucher disease is a genetic disorder where lipids build up in cells and certain organs, causing problems for the patient. Type 1 patients often live well into adulthood, but life expectancy is much lower for patients with the rarer type II and type III forms of the disease.
Genzyme are hoping to market their twice-daily oral therapy for patients with Type I Gaucher’s, which occurs in roughly 1 in 50,000 live births, in order to offer a more convenient substitute to their current standard-of-care treatment, Cerezyme (imiglucerase), which is given via intravenous infusions.
Cerdelga is a novel ceramide analogue designed to partially inhibit the enzyme glucosylceramide synthase and thereby slash the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher. It therefore differs from current treatments on the market, which generally focus on lengthy enzyme replacement therapy as a way to keeping Gaucher under control.
Phase III clinical studies of the drug, part of the largest clinical trial programme ever focused on Gaucher which involved 400 patients who were treated across 30 countries, showed non-inferiority with Cerezyme in addition to an improvement in spleen size.
If approved, Cerdelga will be vying for market share with Shire’s Vpriv and Pfizer/Protalix Biotherapeutics’ Elelyso, although unlike Cerdelga these are both injected. Actelion also have an oral drug on the market for type I Gaucher, Zavesca (miglustat), but this is only used in people who cannot be treated with enzyme replacement therapy.