from Wells Fargo **Today, Protalix, and partner Pfizer, announced that the FDA has requested additional data regarding the Chemistry, Manufacturing and Controls (CMC) section of the New Drug Application (NDA) for Uplyso (taliglucerase alfa) for the treatment of Gaucher disease prior to accepting the filing and establishing a PDUFA date. The request centered predominately on validation of the manufacturing process in the company's scaled-up manufacturing facility a process which has been established and reviewed by the FDA. Protalix completed its rolling NDA filing for Uplyso (taliglucerase alfa) on December 9, 2009, suggesting an early June 2009 PDUFA and approval. This delay will likely push approval back until Q4 2010 as Protalix will submit the requested data to the FDA in Q2 2010. This is an incremental positive for Genzyme.
**Protalix's taliglucerase lacked the initial penetration/ capacity (~100 patients) of Shire's velaglucerase. Recall Shire has capacity to treat and 300-600 Gaucher patients in 2009 and up to around a total of 900 in 2010. However, according to a U.S. clinical investigator who treats a wide spectrum of rare genetic disorders and involved in the velaglucerase and GENZ-112638 Ph. III programs; due to the lengthy IRB process, Gaucher patients were not able to access velaglucerase until December thus most waited for Cerezyme. Of 45 Gaucher patients treated at the center, only 3 opted to participate in the Shire program and likley not to return to Cerezyme. The center opted not to participate in the Protalix program (Uplyso). Through conversations with other U.S. colleagues the clinician felt their Gaucher experience was at worse similar to other clinics (i.e., a only few patients switching off Cerezyme for good and some not accessing velaglucerase at all).
***Recall on Monday November 23 Genzyme began shipping Cerezyme. Genzyme released the guidance for the Gaucher community on restarting Cerezyme to treating clinicians on November 24, 2009 outlining basic details on the release of Cerezyme and Genzyme made patients aware of the new product shipments on November 25. Initially, Cerezyme was available for the most vulnerable patients. On December 28, 2009 Genzyme began making Cerezyme available for all patients. This reintroduction enabled Cerezyme to be available for two months prior to the approval of Shire's velaglucerase alfa NDA which has been given a PDUFA of February 28, 2010 and is manufactured using a mammalian vector.
