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news.Retrophin has received orphan drug designation from the European Commission (EC) for Sparsentan to treat focal segmental glomerulosclerosis (FSGS).
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Sparsentan, also known as RE-021, is an investigational therapeutic agent which acts as a potent angiotensin receptor blocker, and a selective endothelin receptor antagonist, with in vitro selectivity toward endothelin receptor type A.
Retrophin is currently evaluating Sparsentan in the DUET Phase 2 clinical study. The company said the data obtained from the trial may support an application for accelerated approval of the drug on the basis of proteinuria as a surrogate endpoint.
Retrophin executive vice president and head of research & development Alvin Shih said: "Obtaining orphan designation for sparsentan in the European Union is an important milestone for both Retrophin and the FSGS patient community.
"There is a clear unmet medical need in FSGS. As part of our ongoing commitment to developing treatment options for patients suffering from rare diseases like FSGS, we look forward to completing the DUET trial and reading out results in 2016."
Earlier this year, the US Food and Drug Administration granted orphan drug designation to Sparsentan for the treatment of FSGS, which is a cause of nephrotic syndrome in children and adolescents, as well as an leading cause of kidney failure in adults.
Ligand Pharmaceuticals licensed global rights of Sparsentan to Retrophin in 2012.