Chorioretinal atrophy developed in over a quarter of eyes after gene therapy injection
Interim Results From a Multicenter Observational Safety Study of Patients Treated With Voretigene Neparvovec-rzyl in the United States.
eyes developed patchy thinning/scarring of the retina's back layers, worse than before
With a median 3.7 years of follow-up (range, 2.9-4.9), most patients (95%) reported ≥1 TEAE (387 events); 67% were procedure related, 9% were attributed to VN, and 82% were mild.
The area of CRA mostly localized to the retinotomy or bleb area, with 40% of eyes with CRA having atrophy outside the bleb area and highly correlated between fellow eyes (r=0.97; P<0.0001).
CRA risk was higher with automated foot pedal administration of VN (RR, 5.33; 95% CI, 1.27-22.40) and in patients with myopia (RR, 8.40; 95% CI, 1.16-60.69).
BCVA and FST were comparable between CRA and non-CRA eyes, although a subset of CRA eyes showed greater FST improvement at most time points up to Year 3.
This interim analysis supports the long-term safety of VN in a real-world US cohort, consistent with clinical trial results.
CRA did not appear to diminish sustained functional and anatomical benefits with a minimum follow-up of nearly 3 years.
Ongoing data collection will further clarify the occurrence, impact, and risk factors for CRA while continuing to monitor VN safety in clinical practice.