RARE Daily

FDA Grants Fast Track to Aprinoia’s Diagnostic for Progressive Supranuclear Palsy

May 20, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Aprinoia Therapeutics’ APN-1607, a positron emission tomography tracer for imaging tau protein in patients with suspected progressive supranuclear palsy.

Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder caused primarily by the accumulation of a specific form of tau in subcortical brain regions. There are no FDA-approved diagnostic markers for PSP or any other rare tau-related disorders such as frontotemporal dementia, and until now, diagnosis has primarily relied on clinical assessment. APN-1607 may enable more accurate diagnosis at earlier disease stages, potentially improving patient management and resulting in more efficient clinical trial designs for novel therapies.

APN-1607 is a radioactive fluorinated molecule developed to visualize and quantify 3R and 4R tau aggregates by PET imaging across a number of diverse tau-related disorders, including PSP, frontotemporal dementia, and Alzheimer’s disease, among others. APN-1607 has been clinically utilized in more than 3,000 patients through investigator-initiated and sponsor trials, supporting its potential clinical utility as a diagnostic marker for tau-related disorders. In December, Aprinoia received a nod from the FDA to conduct a global phase 3, multicenter, open-label study to evaluate the efficacy and safety of APN-1607 as a diagnostic marker in patients suspected of having PSP. Aprinoia previously received an Orphan Drug designation from the FDA for APN-1607 as a diagnostic agent for PSP.

“APN1607 is a unique imaging agent as it was designed to detect specific forms of tau implicated in PSP and other related disorders. Sadly, patients with PSP can remain undiagnosed for several years as it is often confused with other Parkinson’s like disorders, especially during the early stages,” said Brad Navia, chief medical officer of Aprinoia Therapeutics. “If approved, APN-1607 would provide physicians with an important diagnostic tool that will allow them to diagnose PSP with greater confidence and differentiate it from other disorders, thereby improving the management of these patients.”

Fast Track designation is designed to facilitate the development and expedite the review of product candidates that demonstrate the potential to address an unmet medical need, with the goal of advancing important new diagnostic and treatment options to patients more quickly than traditional regulatory routes. It allows for early and frequent communication with the FDA, including discussions around the product candidate’s development plan and regulatory review process are ensured. If the relevant criteria are met, the product candidate may be eligible for Accelerated Approval and Priority Review.

Photo: Brad Navia, chief medical officer of Aprinoia 

Stay Connected

Sign up for updates straight to your inbox.