Sanofi (France)'s Gaucher Pill Meets Goal in Phase 3 Trial

Sanofi's experimental pill to treat Gaucher disease, a rare inherited condition, has met its main goal in a late-stage trial, the French drugmaker said on Tuesday. Patients treated with the drug, eliglustat tartrate, showed a statistically significant improvement in the size of their spleen after nine months compared with those who took a placebo, Sanofi said in a statement. The treatment was well tolerated and showed no serious side effects during the trial, it said. Gaucher disease is a rare genetic disorder affecting some 10,000 patients worldwide. Patients with the disease are deficient in an enzyme that breaks down a certain type of fat, leading to potentially life-threatening organ damage and bone problems.

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