Yesterday, Shire plc (SHPGY) submitted its new drug application (NDA) for velaglucerase alfa, its enzyme replacement therapy for the treatment of type 1 Gaucher disease, with the U.S. Food and Drug Administration (FDA).  

Shire also announced that velaglucerase alfa achieved its primary and secondary endpoints in all three of its phase III studies, including a head-to-head study with Genzyme Corp.’s (GENZ) Cerezyme. Another study, assessing the safety of switching patients from Cerezyme therapy to velaglucerase alfa, also posted positive results.

We were pleased to see Shire submit the NDA slightly ahead of schedule. The European submission is scheduled to take place by year’s end.

Once approved and launched commercially, velaglucerase alfa will compete directly with Cerezyme. Shire is currently providing the treatment free of cost to patients. As a reminder, Genzyme is facing problems with the supply of Cerezyme due to the temporary shutdown of its Allston manufacturing plant. While manufacturing has resumed, Genzyme will not be in a position to supply new lots of Cerezyme until later this year.

In order to make up for the shortage in supply of Cerezyme, the FDA had approached Shire and another company, Protalix Biotherapeutics Ltd., to come up with treatment protocols for their Gaucher disease candidates. The FDA accepted both treatment protocols, which means that the drugs will be available for the treatment of Gaucher patients prior to their commercialization.

We believe the temporary availability of velaglucerase alfa will give physicians the opportunity to become familiar with the product prior to its commercial launch. Moreover, positive results from the head-to-head study with Cerezyme could help velaglucerase alfa gain share from Cerezyme.

A response from the FDA on the approval status of velaglucerase alfa should be out in the first half of 2010.
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