FDA selects Rett syndrome gene therapy for rare disease pilot drug program

The FDA has selected an investigational AAV9 gene therapy for Rett syndrome to participate in a pilot program created to provide a boost to novel drug development for rare diseases, according to a release from the manufacturer.

The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program will allow the company to engage in enhanced communications with the FDA to accelerate the pace of development for NGN-401, as well as to address product-specific development issues, including clinical study design, choice of control group and choice of patient population, Neurogene Inc. said in a press release.

NGN-401 is currently being evaluated in two doses within a phase 1/2 clinical trial to assess safety, tolerability and preliminary efficacy of a one-time intracerebroventricular administration in female pediatric patients with Rett syndrome.

NGN-401 previously received orphan drug, fast track and rare pediatric designations from the FDA.

“We are honored that NGN-401 gene therapy for Rett syndrome has been chosen as one of only three CBER programs for FDA’s START pilot program and are grateful that the FDA has committed to investing significant agency resources to accelerate development of NGN-401,” Neurogene founder and CEO Rachel McMinn, PhD, said in the release. “We look forward to participating in this landmark effort with the FDA as we seek to rapidly advance NGN-401 toward a potential registrational study.”

Neurogene additionally said in the release it would report interim efficacy data from the study during the fourth quarter of 2024.