Ups and Downs of Gene Therapy Weighed by FDA’s Marks

Aggressively using all the FDA’s regulatory flexibility to advance burgeoning treatments for rare diseases, particularly gene therapy, was a theme highlighted Monday by CBER Director Peter Marks.

Speaking at a webinar sponsored by the Alliance for a Stronger FDA, Marks addressed a range of initiatives including his department’s balancing act to move forward on approving potentially lifesaving products while ensuring total safety and effectiveness.

Marks believes the advantages shown so far in gene therapy treatments are enough to move forward aggressively, highlighting the 15 currently approved for use in the U.S. And while admitting that the pace of proven successes may not be as quick as he’d like, Marks made a point to highlight Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy for spinal muscular atrophy approved in 2019 that has shown remarkable success when administered to infants during the first six months of life. “When given to children who would normally be dead by age three… these children walk, run and appear to develop normally.” While not perfection, he said, it presents, in today’s world, “….a very good gene therapy approach.”

To read the whole story, click here to subscribe.

Related Topics