Regulatory innovations will speed ultra-rare therapies, Marks says

In an interview with The BioCentury Show, Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, articulated a vision for using a suite of regulatory tools to smooth the path for medicines to treat ultra-rare diseases. He envisions accelerated approval facilitating the development of gene therapies, a new platform technology pathway streamlining gene therapy development, and a pilot program that applies lessons learned from COVID-19 vaccine development to speeding the development of orphan drugs.

The interview also touched on pandemic preparedness, the future of COVID-19 vaccines, and CBER’s coordination with other parts of FDA. ...