RT001 Gets Orphan Drug Designation in Progressive Supranuclear Palsy


Retrotope had already received FDA approval to test RT001, a chemically modified polyunsaturated fatty acid agent, in expanded access trials of 3 patients with progressive supranuclear palsy.

Peter Milner, MD

Peter Milner, MD

According to an announcement from Retrotope, the FDA has granted orphan drug designation to the company’s investigational drug RT001 for the treatment of patients with progressive supranuclear palsy (PSP). The FDA’s Division of Neurology Products had previously granted approval to Retrotope to test RT001 in expanded access trials of 3 patients with PSP.1

“PSP is a disease involving modification and dysfunction of tau protein. RT001’s mechanism of action both lowers lipid peroxidation and prevents mitochondrial cell death of neurons which is associated with disease onset and progression,” Peter Milner, MD, chief medical officer of Retrotope, said in a statement. “Also, RT001 has a synergistic downstream benefit in the pathophysiology of PSP. This orphan designation encourages clinical trials and rapid review of data from trials to treat this intractable disease.”

RT001 is a chemically stabilized fatty acid that incorporates into mitochondrial and cellular membranes and, in turn, stabilizes them. The patented, first-in-class, orally available isotopic-reinforced polyunsaturated fatty acids (D-PUFA) has been shown to decrease levels of lipid peroxidation in PSP patient mesenchymal stem cells, as well as restore mitochondrial function and structure to damaged cells.

Researchers at Retrotope expect the drug to lower the amount of lipid peroxidation, restore normal mitochondrial function and prevent mitochondrial cell death once distributed as an essential fat throughout tissues of the body.

In January 2020, the company announced the first patient dosing of RT001 in Friedreich’s ataxia (FA). The randomized, double-blind, placebo-controlled ongoing phase 2/3 clinical trial is evaluating the efficacy, safety, and tolerability of RT001 in subjects with FA.2

At the 2019 American Academy of Neurology (AAN) annual meeting, the Retrotope presented positive findings from its expanded access trial in a patient with late onset Tay-Sachs disease (LOTS). The 44-year-old patient with LOTS demonstrated improvements in a variety of patient outcomes (choking coughs, strengths falls), as well as objective measures of performance (timed walks, quantified speech measures, Archimedes spiral).3

“We want to sincerely thank the FDA’s OOPD for this designation which allows us accelerated review and more flexibility in pursuing this indication. We are also grateful to the researchers, patients and clinicians whose work contributed to the results supporting our filing an investigational new drug [IND] application to FDA in this terrible disease,” Robert Molinari, PhD, CEO of Retrotope, said in a statement.


1. US FDA grants orphan drug designation for Retrotope’s RT001 in the treatment of progressive supranuclear palsy (PSP)[news release]. Los Altos, CA: Retrotope. February 18, 2020. globenewswire.com/news-release/2020/02/18/1986602/0/en/US-FDA-Grants-Orphan-Drug-Designation-for-Retrotope-s-RT001-in-the-Treatment-of-Progressive-SupraNuclear-Palsy-PSP.html. Accessed February 20, 2020.

2. Retrotope expands its drug pipeline with the first dosing of RT001 in patients with Friedreich’s ataxia (FA) three clinical trials centers now open for enrollment; two additional ones initiating [news release]. Los Altos, CA: Retrotope. January 9, 2020. biospace.com/article/releases/retrotope-expands-its-drug-pipeline-with-the-first-dosing-of-rt001-in-patients-with-friedreich-s-ataxia-fa-three-clinical-trials-centers-now-open-for-enrollment-two-additional-ones-initiating/. Accessed February 20, 2020.

3. Retrotope announces RT001 clinical data presentation at the 2019 American Academy of Neurology annual meeting [news release]. Los Altos, CA: Retrotope. May 6, 2020. globenewswire.com/news-release/2019/05/06/1817720/0/en/Retrotope-Announces-RT001-Clinical-Data-Presentation-at-the-2019-American-Academy-of-Neurology-AAN-Annual-Meeting.html. Accessed February 20, 2020.

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