
Spanish pharmaceutical company Ferrer’s FNP-223 has obtained fast track designation from the US Food and Drug Administration (FDA) for its potential in treating progressive supranuclear palsy (PSP).
FNP-223 is an in-licensed product from Swiss biotechnology firm Asceneuron. The licensing deal was signed in February 2023.
Presently, FNP-223 is undergoing a Phase 2 clinical trial. The study aims to assess its safety, efficacy, and pharmacokinetics in adult patients with possible or probable PSP-Richardson syndrome, a common form of this neurodegenerative condition.
FNP-223 is administered orally and acts as a reversible and substrate-competitive inhibitor of the O-GlcNAcase enzyme.
The compound binds to the enzyme’s active site, obstructing the substrate’s access to the catalytic pocket, which in turn prevents the removal of O-GlcNAc modifications from proteins like tau. This inhibition is anticipated to increase glycosylated tau proteins, reducing abnormal tau aggregation into neurofibrillary tangles over time.
Ferrer CEO Mario Rovirosa said: “We are thrilled to receive fast track designation from the FDA for FNP-223 in the treatment of PSP. Consistent with our purpose of using business to fight for social justice, we are committed to advancing this promising therapy as quickly as possible to benefit as many patients as possible.”
According to Ferrer, the fast track status granted by the FDA is a crucial step in the development of FNP-223. This designation facilitates more frequent interaction with the FDA and opens pathways for accelerated approval and priority review if certain conditions are satisfied.
Progressive supranuclear palsy affects patients with symptoms such as speech difficulties, balance issues, altered gait, and cognitive challenges. The disorder is estimated to affect approximately five individuals per 100,000 people, predominantly those over 60 years of age.
The disease is linked to the abnormal build-up of tau proteins in specific brain regions leading to neurodegeneration. Preclinical studies have shown that FNP-223 can hinder abnormal tau protein accumulation in neurons.