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Sanofi (SNY - Analyst Report) recently announced encouraging data from two phase III studies (ENGAGE and ENCORE) evaluating the use of its oral candidate, eliglustat tartrate, for Gaucher disease type I.   

The randomized, double-blind, placebo controlled ENGAGE phase III study enrolled 40 treatment-naïve patients with Gaucher disease type I. Results from the study revealed a statistically significant improvement in spleen size at nine months for patients treated with eliglustat tartrate compared to those in the placebo arm.

Additionally, improved hemoglobin levels, platelet levels and liver volumes were observed in patients in the eliglustat tartrate arm compared to patients treated with placebo.

Sanofi also reported data from the second phase III study, ENCORE. The randomized, open-label study enrolled 160 adults with Gaucher disease type I. The study compared eliglustat tartrate with Sanofi’s Cerezyme, the current standard of care for Gaucher disease type I. Eliglustat tartrate demonstrated non-inferiority to Cerezyme and met the primary endpoint of the study.

Our Take

Eliglustat tartrate, if approved, will provide a convenient treatment option to Gaucher disease type 1 patients as compared to Cerezyme, which is administered through intravenous infusions. We note that Sanofi is looking to develop its pipeline to combat the generic threat that is faced by several of its products.

Sanofi carries a Zacks Rank #3 (Hold) in the short run. Pharma companies that currently look better-positioned include Astellas Pharma (ALPMY), Eli Lilly and Company (LLY - Analyst Report) and Bayer (BAYRY - Analyst Report). All three companies carry a Zacks Rank #2 (Buy).

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