Retrophin Receives European Orphan Drug Designation for RE-024 for the Treatment of Pantothenate Kinase-Associated Neurodegeneration

Retrophin, Inc. (NASDAQ:RTRX) announced that the European Commission has granted orphan drug designation to RE-024, the Company’s novel investigational phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration (PKAN), a rare and life-threatening genetic disorder with no approved treatment option.
According to the company’s news:

In the European Union (EU), orphan drug designation is granted to a medicinal product intended to treat a life-threatening or chronically debilitating rare disease with no approved treatment option. In the EU, a disease is designated as rare if it affects no more than five in 10,000 people. In addition to a 10-year period of EU market exclusivity upon product approval, orphan drug designation provides fee waivers, protocol assistance, and single marketing authorization under the centralized procedure that is valid in all EU countries.

Alvin Shih, M.D., executive vice president and head of research & development for Retrophin commented:

Receiving orphan drug status in the EU is another encouraging step in the development of RE-024 for the treatment of PKAN. Without an approved therapy, many people living with PKAN currently face a significant burden of illness. We look forward to continuing our development of RE-024 with the ultimate goal of delivering an effective therapeutic option to these patients.

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