FDA plans predictable approval pathway for ultrarare genetic diseases

FDA has announced a new regulatory pathway aimed at facilitating development of therapies for ultrarare genetic disorders. Building on principles outlined during the Biden administration, the pathway seeks to provide clarity and predictability about approvable endpoints for conditions that can’t be evaluated using standard clinical trial designs.

Separately, the agency has provided some additional information about the forthcoming “plausible mechanism pathway” for the rarest of ultrarare disease therapies. The pathway seems to be tailored to facilitate approvals of technologies such as antisense oligonucleotides (ASOs) used to create n-of-1 treatments. It could speed the development of the kinds of treatments the n-Lorem Foundation develops and the N=1 Collaborative is trying to advance. ...