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EMA Grants Orphan Medicinal Product Designation for Retrotope's RT001 in the Treatment of Infantile Neuroaxonal Dystrophy (INAD), a PLA2G6 associated neurodegeneration (PLAN)LOS ALTOS, Calif., March 13, 2019 (GLOBE NEWSWIRE) -- Retrotope received notification from the Committee for Orphan Medicinal 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. Robert Molinari, Ph.D. CEO of Retrotope commented: “We want to sincerely thank the European Commission for granting us orphan status in such a devastating, fatal, and untreated disease. We are also grateful to the researchers, patients and clinicians whose work contributed to the results supporting our orphan status, and hope our currently-enrolling late stage clinical trial in this ultra-rare condition can recapitulate the favorable results we’ve seen in two Expanded Access patients.” Peter Milner, MD, Chief Medical Officer of Retrotope, added, “Patients from all over the world are enrolled in our open-label, US trial in INAD. We look forward to be able to treat patients as quickly as possible world-wide if our current US pivotal trial achieves positive results. While not the subject of our current trials, the INAD genotype includes classical INAD, non-infantile onset or Atypical Neuroaxonal Dystrophy (ANAD), and PLA2G6 deficiency induced young-onset Parkinson’s disease (YOPD).”
INAD is an ultra-rare, devastating life-shortening neuro-degenerative disorder that affects only a few hundred patients worldwide. Infants with INAD appear to develop normally until approximately 14 to 18 months of age, when they begin to experience progressive mental and psychomotor development declines. Life threatening complications typically develop by the end of the first decade, and there is no approved treatment. RT001 is a chemically stabilized fatty acid drug that confers resistance to lipid peroxidation in mitochondrial and cellular membranes, a pathology found to be a causative element of cell death in disease models. About RT001 About Retrotope SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022
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