STAT+: As its immunology work draws attention, Sanofi also pushes forward in rare disease

Sanofi’s Cerezyme has long been used as a treatment for Gaucher disease, a rare illness that can cause enlarged spleens and livers, bone pain, and fatigue. Patients with the kind of Gaucher that causes neurological problems, however, haven’t had a treatment option to address their symptoms. 

But on Monday, the French pharma firm reported that clinical trial results showed its experimental oral drug venglustat improved the neurological manifestations of what’s known as type 3 Gaucher disease, which can include poor muscle coordination and cognitive issues. Sanofi now plans to submit the drug to regulators. 

“For many years now, enzyme replacement therapy has been very good for the peripheral symptoms of Gaucher,” said Houman Ashrafian, the company’s head of R&D, referring to drugs like Cerezyme that work by effectively delivering an enzyme that patients with Gaucher can’t make on their own. “However, the neurological progressive features of Gaucher … had not been very well cared for.”Continue to STAT+ to read the full story…