The Food and Drug Administration (FDA) has granted Orphan Drug designation to RT001 (Retrotope) for the treatment of progressive supranuclear palsy (PSP).

PSP is a rare, progressive neurodegenerative disorder that affects movement, gait and balance, speech, swallowing, vision, behavior and thinking. While the exact cause of the disorder is unknown, it is considered a tauopathy, which is characterized by the accumulation of tau protein in the brain leading to neuronal death and dysfunction.  

RT001 is a first-in-class, oral deuterated polyunsaturated fatty acid (D-PUFA) that stabilizes mitochondrial and cellular membranes by “fireproofing” against reactive oxygen species. The investigational drug prevents lipid peroxidation caused by polyunsaturated fats (PUFAs), which is believed to be the primary source of several degenerative diseases (eg, Parkinson and Alzheimer disease). The designation is supported by preclinical data that demonstrated the ability of RT001 to inhibit lipid peroxidation, thereby protecting mitochondrial function. 

“RT001’s mechanism of action both lowers lipid peroxidation and prevents mitochondrial cell death of neurons which is associated with disease onset and progression,” said Peter Milner, MD, Chief Medical Officer of Retrotope. “Also, RT001 has a synergistic downstream benefit in the pathophysiology of PSP.”

Related Articles

The Company is also investigating RT001 for the treatment of infantile neuroaxonal dystrophy (phase 2/3 study) and Friedreich ataxia (phase 3 study).

For more information visit