"Sparsentan has the potential to be a significant medicine, and could transform the treatment of FSGS," said John Higgins, President and Chief Executive Officer of Ligand Pharmaceuticals.
"The team at Retrophin continues to make great progress advancing the program, and obtaining orphan designation provides an additional layer of potential market exclusivity. This is a valuable late-stage asset in our broad portfolio of over 100 shots-on-goal."
Higgins added, "Late last year, Ligand highlighted a selection of major pipeline programs that we called the Big Six for investors to follow in 2015.
"In recent weeks, three of the six programs have had positive developments with the NDA submission for Captisol-enabled Melphalan, positive Phase 3 data for Captisol-enabledDelafloxacin IV, and now orphan designation for an important drug targeting a rare disease with major unmet medical need. We are pleased with the momentum we have for these key programs early in the year."
Sparsentan is an investigational therapeutic agent which acts as both a selective endothelin receptor antagonist and an angiotensin receptor blocker. Retrophin is conducting the Phase 2 DUET trial of Sparsentan for the treatment of FSGS, a leading cause of end-stage renal disease. There are currently no therapies approved for the treatment of FSGS in the United States.
Ligand licensed worldwide rights of Sparsentan (RE-021) (formerly known as DARA) to Retrophin in 2012, at the time of Retrophin’s formation.