EMA Grants Orphan Medicinal Product Designation for Retrotope 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.
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 – Retrotope received 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.
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.
RT001 is a patented, first-in-class, orally available D-PUFA, a deuterated polyunsaturated fatty acid, that incorporates into mitochondrial and cellular membranes and stabilizes them. Retrotope and others have discovered that lipid peroxidation, the free-radical damage of polyunsaturated fats (PUFAs) in mitochondrial and cellular membranes, may be the primary source of cell death in several degenerative diseases, including Friedreich’s Ataxia (FA) and INAD. The presence of D-PUFAs (RT001) can help protect (“fireproof”) against this attack and potentially restore cellular health.
Retrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease-modifying therapies for many intractable diseases, such as Parkinson’s, Alzheimer’s, INAD, ALS, Friedreich’s ataxia (FA), Late
Onset Tay Sachs (LOTS), mitochondrial myopathies, and retinopathies. RT001, Retrotope’s first lead candidate, is being tested in controlled trials in INAD and Friedreich’s ataxia. For more information about Retrotope, please visit www.retrotope.com.
SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022