Frontotemporal dementia drug gets fast track designation from FDA

The FDA has cleared an investigational new drug application for AVB-101 to treat those with frontotemporal dementia with mutations in the progranulin gene, according to a release from the manufacturer.

AVB-101 is designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to areas of the brain affected by frontotemporal dementia (FTD), AviadoBio said in the release.

The FDA additionally granted the novel therapeutic fast track designation, which aims to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need.

FTD is a form of early-onset dementia that typically leads to death within 7 to 13 years of symptom onset. Those with the condition typically experience a rapid change or decline in personality, behavior, language, emotional regulation and reduced mobility.

A leading cause of dementia in people aged younger than 65 years with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people, genetic FTD accounts for roughly one-third of all cases and is most frequently associated with autosomal dominant mutations in three genes, including the progranulin, according to the release.

“The IND clearance and fast track designation of AVB-101 are important milestones for the FTD community and for our company,” AviadoBio CEO Lisa Deschamps said in the release. “Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations.”

Both the FDA and European Commission granted orphan designation to AVB-101 for the treatment of FTD in 2022. AviadoBio recently announced enrollment is open in European countries for ASPIRE-FTD, an open-label, multi-center dose-escalation study designed to evaluate the safety and preliminary efficacy of the treatment in patients with FTD-GRN.