Shire goes for early approval in Gaucher disease
pharmafile | September 1, 2009 | News story | Research and Development, Sales and Marketing | Gaucher's, Shire
Shire has filed for early US approval of its Gaucher disease drug velaglucerase alfa, as supplies to Genzyme's established rival treatment start to run out.
Shire announced the filing along with positive phase III results, and has raced for approval since a shortage of treatment options was caused by a cut in supply to Genzyme's Cerezyme, caused by a contamination in its production plant.
An early FDA approval for Shire will allow physicians to prescribe its treatment sooner than anticipated, and present an opportunity for early exposure in the US.
Sylvie Grgoire, president of Shire human genetic therapies, said: "The submission of the New Drug Approval for velaglucerase alfa, earlier than previously announced, is an important milestone for Shire, bringing us another step closer to providing a new treatment option for patients with Type 1 Gaucher disease."
Velaglucerase alfa is an enzyme replacement therapy for the treatment of Type 1 Gaucher disease. The disease results from a deficiency of the enzyme glucocerebrosidase in the body, caused by a genetic mutation received from both parents. The disease course is quite variable, ranging from no outward symptoms to severe disability and death.
The contamination problems at Genzyme's manufacturing plant have been an opportunity for rival companies developing treatments for Gaucher and Fabry disease, including Shire, Amicus Therapeutics and Protalix.
The problems prompted the FDA in July to say it would fast-track access to rival products to maintain treatment supplies to patients. Mindful of the impending shortages from Genzyme, it asked Shire and Protalix to file treatment access protocols for their products in the hope that they can be made available to patients ahead of marketing approval.
Shire then filed a treatment protocol for its drug candidate two months ago, saying it would provide the drug free of charge initially, in order to provide access to patients as quickly as possible.
New results
The company's full submission for marketing approval comes as it announces positive results from the final two Phase III studies of the drug, with both studies reaching all of their primary and secondary endpoints.
The product was generally well tolerated in both treatment of nave and previously treated Gaucher patients.
Grgoire said: "We are also pleased to report that the data from our 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 Phase III trial. In addition, we are on-track to submit our European filing for velaglucerase alfa by the end of 2009."
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