Shire has submitted an earlier-than-expected application to US regulators for its new Gaucher disease drug at a time when patients are looking to cope with a potential shortage of the only approved drug for the disease, Genzyme Corp's Cerezyme.

Last month, Shire announced the start of a rolling submission to the US Food and Drug Administration on the basis of data from the first of three Phase III studies of velaglucerase alfa, for the treatment of type 1 Gaucher disease. It has now announced positive results from the final two late-stage studies of the drug and filed a New Drug Application with the agency.

Sylvie Gregoire, president of Shire Human Genetic Therapies, said that the data from the final two Phase III trials “met our expectations by reaching all of their primary and secondary endpoints, demonstrating consistency” with the results recently reported from the first late-stage study. In addition, “we are on-track to submit our European filing for velaglucerase alfa by the end of 2009," she added.

Shire said that its velaglucerase alfa programme included “the largest and most comprehensive set of Phase III clinical trials conducted to date for Gaucher disease”. Over 100 patients at 24 sites in ten countries have participated.

Shire is already providing the treatment free of charge in the USA as a result of Genzyme temporarily shutting down its manufacturing facility in Boston in June after a bioreactor was contaminated with a virus. This affected production of Cerezyme and Fabrazyme (agalsidase beta), which is indicated for Fabry disease.

Israeli-headquartered Protalix BioTherapeutics is also hoping to fill the void created by the Cerezyme shortage and it hopes to submit its Gaucher disease treatment prGCD (glucocerebrosidase) by the end of the year.

EMEA concerns over Genzyme facility
Meantime Genzyme noted that inspectors from the European Medicines Agency visited the troubled Allston Landing manufacturing plant in Massachusetts a couple of weeks ago. They identified “no critical observations” but "one major observation and several other observations" at the facility.

Genzyme said it will respond to the inspectors within 15 days “with a plan and timetable for addressing the observations”. The company added that it “also anticipates that the FDA and other regulatory bodies” will inspect the plant.