FDA grants fast track designation to AviadoBio’s dementia gene therapy

07 Nov 2023
www.pmlive.com
Fast TrackOrphan DrugGene TherapyClinical Study
FDA grants fast track designation to AviadoBio’s dementia gene therapy
Preview
Source: PMLiVE
The US Food and Drug Administration (FDA) has granted fast track designation to AviadoBio’s experiential dementia gene therapy and given clearance for the candidate to be studied in a subset of frontotemporal dementia (FTD) patients.
AVB-101 will now benefit from the FDA’s fast track process, which is designed to improve the efficiency of product development and accelerate the review of treatments for serious conditions.
FTD, a form of early-onset dementia, is the leading cause of dementia in people aged under 65 years and typically leads to death within seven to 13 years of symptom onset.
Genetic FTD cases account for about one-third of cases and are most frequently associated with autosomal dominant mutations in three genes, including the progranulin (GRN) gene.
The potential one-time therapy AVB-101 has been designed as to halt disease progression by delivering a functional copy of GRN to restore levels of progranulin to areas of the brain affected by FTD.
AviadoBio’s chief medical officer, David Cooper, said: “Our preclinical programme shows robust bio-distribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects.”
The FDA and the European Commission both granted orphan designation to the candidate as a treatment for FTD in 2022, and AviadoBio recently announced that enrolment to its phase 1/2 of the candidate in patients with FTD-GRN had begun in Europe.
Lisa Deschamps, the company’s chief executive officer, said: “Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with FTD with progranulin mutations.
"Receiving fast track designation underscores the significant need for treatment options for these patients, and we are eager to soon open US clinical trial sites and offer an innovative option for eligible patients with FTD-GRN.”
The FDA’s decision on AVB-101 comes shortly after the Alzheimer's Society raised concerns that not enough people are being diagnosed with young-onset dementia in England.
A report from NHS England revealed that 34,412 people in England have been diagnosed with young-onset dementia, but the Alzheimer’s Society has said that the number of people affected may be closer to 53,206, based on estimates shared with other dementia charities.
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
Drugs
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.