FDA Offers Direction on Trials of Genome-Editing Gene Therapies
Sponsors of gene therapy trials using genome editing (GE) should focus on enrolling patients with severe or debilitating disease, according to a draft FDA guidance that also advises long-term follow-up of trial participants.
In the guidance — which addresses product development and preclinical considerations as well as clinical trial design — the FDA recommends that trials employing GE only enroll patients who have no other treatment options because these patients may be more willing to accept the risks of an investigational GE drug. However, they may also be more prone to a higher number of adverse events or be on additional medications, the agency warns.
For pediatric trials associated with greater-than-minimal risk, sponsors should initially enroll at least one cohort of adult participants, whenever possible, to generate preliminary safety, feasibility and efficacy data that support pediatric enrollment. When enrollment of pediatric patients is justified, the FDA recommends that sponsors enroll adolescents prior to enrolling younger children and infants.
Feedback on the draft guidance can be submitted at any time, according to the FDA.
Read the full guidance here: https://bit.ly/3qgDups.