19 May FDA ‘leans in’ to accelerated approval for rare disease drugs

CBER director says ‘moment is tender for gene therapy,’ sees opportunity to ‘salvage’ treatments

By Kristina Fiore, MedPage Today

The FDA is “leaning in” to cell and gene therapies, expressing a willingness to use the accelerated approval pathway for these products, particularly as new leadership is set to take charge of a recently revamped office.

That was the tone of two talks given by Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) at the FDA, during the American Society of Gene & Cell Therapy (ASGCT) meeting in Los Angeles this week. CBER oversees the regulation of cell and gene therapies via its newly created “super office,” the Office of Therapeutic Products, which resulted from the reorganization of the former Office of Tissues and Advanced Therapies.

“When you’re dealing with very small populations, we’re probably willing to use some regulatory flexibility there … with the note that we will have to have the confirmatory data at some point,” Marks said during one of the talks.

“It’s not about lowering the standard,” he emphasized. “It’s about fixing the environment so that we are able to provide the certainty such that people will bring things forward.”

Marks’ office has paid particular attention to rare disease therapies in recent months, noting that FDA plans to launch an Operation Warp Speed-style pilot program for bringing these treatments to market faster. In 2022, he said, the agency approved five cell and gene therapies, and hopes to increase that tally in the coming years. Read more …