FDA grants orphan drug designation for frontotemporal dementia therapy

The FDA and European Commission have both granted orphan drug designation to AviadoBio for its new gene therapy to treat frontotemporal dementia, according to a company press release.

“Achieving orphan designations in both the U.S. and E.U. represents an important milestone in bringing AVB-101 to FTD patients who are living with a debilitating disease without treatment options. We are committed to advancing our proprietary gene therapy platform and working with regulatory authorities and the FTD community to bring a desperately needed option to patients,” Lisa Deschamps, CEO of AviadoBio, said in the release.

AVB-101 is an investigational, adeno-associated virus gene therapy designed for patients with FTD with mutations in the progranulin gene. The drug is designed to slow or arrest disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore normal progranulin levels.

Deschamps added that she anticipates initiating clinical trials later in 2022.

“As a clinician who has been treating this patient group for many years, I am looking forward to seeing how this therapeutic candidate performs in clinical studies,” Christopher Shaw, MD, co-founder and chief scientific and clinical adviser at AviadoBio, stated in the release.