EMA Grants Orphan Medicinal Product Designationfor Retrotope’s RT001 in the Treatment of Infantile Neuroaxonal Dystrophy (INAD), a PLA2G6 associated neurodegeneration (PLAN)
Retrotope received today a notification from the Committee for Orphan Medical Products of the European Medicines Agency (EMA) that its request for orphan medicinal product designation for the treatment of INAD with RT001, a chemically-fortified polyunsaturated fatty acid drug, has been granted by the European Commission. Retrotope is currently enrolling a Phase 2/3 trial in the US for INAD, an ultra-rare, severe childhood neurodegeneration due to an inborn error in PLA2G6. RT001 is the first-in-class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage resulting in cell death that is a hallmark of numerous neurodegenerative diseases including INAD.
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