Feb. 18, 2020: Retrotope announced that the U.S.FDA Office of Orphan Products Development granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of Progressive SupraNuclear Palsy (PSP).
Physicians collaborating with the Retrotope have earlier received approval from the FDA’s Division of Neurology Products to test RT001 in Expanded Access trials of three patients having PSP, an orphan neurodegenerative disorder that causes progressive impairment of the balance and walking; impaired eye movement, abnormal muscle rigidity; dysarthria; and dysphagia and eventual death.
PSP is a serious neurodegenerative disease that intensely affects the quality and length of life in adults. Patients are usually disabled within 3-5 years of disease onset. It affects an approximate 17,500 adults in the US. The exact cause of PSP is not known, but it is a form of tauopathy, in which abnormal phosphorylation and accumulation of the protein tau in mid brain leads to destruction of the vital protein filaments in nerve cells, causing their death. Most cases appear to be irregular as an acquired tauopathy and there is no approved drug therapy. A regionally specific increase in the lipid peroxidation has been observed in PSP and mitochondrial structures and functions are compromised and leads to profound oxidation damage.
RT001 is a chemically stabilized fatty acid that, via a novel mechanism, confers resistance to lipid peroxidation in mitochondrial and cellular membranes. In PSP patient mesenchymal stem cells, RT001 has been shown to decrease lipid peroxidation levels and restore mitochondrial structure and function to damaged cells. As RT001 is distributed across human tissues as an essential fat, it is expected to decrease the amount of lipid peroxidation, restore normal mitochondrial function and prevent mitochondrial cell death.https://fda.einnews.com/pr_news/510001029/us-fda-grants-orphan-drug-designation-for-retrotope-s-rt001-in-the-treatment-of-progressive-supranuclear-palsy-psp
