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GEN News Highlights : Mar 1, 2010

Shire’s Gaucher Disease Therapy Gets FDA Green Light

Firm plans to provide easy-access route to therapy and undercut Cerezyme in pricing.

FDA cleared Shire’s Gaucher disease enzyme replacement therapy (ERT), VPRIV™ (velaglucerase alfa for injection). The drug is a human cell line derived hydrolytic lysosomal glucocerebroside-specific enzyme, indicated for use as long-term ERT in adult and pediatric patients with type 1 Gaucher disease.

FDA approval took just six months, following the agency’s designation of VPRIV for priority review. Meanwhile, regulatory review of VPRIV in the EU is ongoing via an accelerated assessment route. Shire anticipates launching the drug in the EU by the end of 2010 and in other countries starting in 2011.

Shire says it plans to price VPRIV 15% lower than the currently available commercial Gaucher disease therapy, Genzyme’s Cerezyme. The firm also aims to make access to VPRIV in the U.S. easier through the implementation of a new Co-Pay Assistance Program within its existing OnePath Access Program. The Co-Pay route is designed to simplify the process and paperwork associated with the initiation of therapy and reduce financial burden on patients, Shire states. Co-Pay will also be available to eligible Hunter syndrome patients requiring therapy with Elaprase®.

Genzyme reported Cerezyme-related revenues of $793,024 in 2009, down from about $1.24 million in 2008. The shortfall relates largely to a four-month halt in production of the drug between June and September 2009, caused by the shut-down of Genzyme’s Allston Landing production plant due to viral contamination. With production now back up and stocks being replenished, Genzyme says patients are expected to be able to resume normal dosing schedules in the first quarter of 2010.

Just last month, Genzyme also said that to more consistently manage the re-supply of Cerezyme to patients worldwide and reduce the interruptions in shipping that occur in the absence of inventory, it is working to immediately build a small inventory buffer. “This buffer will allow a more predictable schedule for Cerezyme delivery through the remainder of 2010 and help avoid the challenges many physicians and patients have experienced in scheduling infusions,” the firm states.

Within the last two weeks Genzyme separately reported positive two-year data from a Phase II trial with its oral type I Gaucher disease candidate, eliglustat tartrate. In 2009, the company confirmed that the Phase II trial had met its primary composite endpoint of a clinically meaningful response in at least two of three endpoints following the initial 52-week study period. These included improvements in spleen size, hemoglobin levels, and platelet levels.

New two-year data from 22 patients continuing with eliglustat therapy showed that spleen volumes decreased from baseline by a mean of 52%, and liver volumes decreased from baseline by 24%. The study data also found hemoglobin levels increased from baseline by a mean of 2.1 grams per deciliter, and platelet counts increased from baseline by a mean of 81%. Additional data from a preplanned analysis suggested that eliglustat therapy over two years may also positively impact on indicators of bone disease, including bone mineral density.

Genzyme is currently enrolling patients into two Phase III trials with eliglustat. ENCORE, is a randomized, open-label study for adult patients with Gaucher disease type 1 and will compare eliglustat tartrate with Cerezyme in patients who have currently been treated using Cerezyme. The Phase III ENGAGE study will evaluate eliglustat in Gaucher disease patients who have received no treatment for their disease over the last 12 months.