PARIS (Reuters) - 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.
Current treatments for the disease include Sanofi’s Cerezyme, an injectable treatment, which for many years dominated the Gaucher market and was at one point the most expensive drug in the world costing more than $200,000 a year.
Full results from the study will be presented at a medical congress in February 2013, Sanofi said.
Reporting by Elena Berton; Editing by Edwina Gibbs