French pharmaceutical group Sanofi said on Tuesday a trial treatment with its Cerdelga drug for patients with type 1 Gaucher disease, a rare genetic disorder, showed promise.
Sanofi said treatment with Cerdelga for nine months resulted in significant improvements in spleen volume, hemoglobin level, liver volume and platelet count compared with a placebo drug.
Results of the randomized clinical trial were published in The Journal of the American Medical Association, Sanofi said.
"We are very encouraged by these results as they ultimately point to a safe and effective oral treatment option for patients living with Gaucher disease," lead author Pramod Mistry, Professor of Pediatrics & Internal Medicine at Yale University School of Medicine, said in a statement.
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