Long-term efficacy and safety of ranibizumab administered pro re nata in Japanese patients with neovascular age-related macular degeneration in the EXTEND-I study.

PURPOSE: To evaluate the long-term efficacy and safety of ranibizumab administered pro re nata (PRN) in Japanese patients with choroidal neovascularization secondary to age-related macular degeneration during the extension phase of the EXTEND-I study.

METHODS: EXTEND-I, an open-label, multicenter, Phase I ⁄ II study comprised: a single-injection (Group A); a multiple-injection (Groups A and B; the latter consisted of patients who did not participate in the single-injection phase); and an extension phase. In the extension phase, a PRN regimen of ranibizumab (0.3 or 0.5 mg) guided by monthly best-corrected visual acuity (BCVA) score and other ophthalmic examinations was employed. The efficacy variables included the mean BCVA change from Month 12 to the last visit in Group B. Safety was assessed in all patients.

RESULTS: In the extension phase, efficacy was assessed only in Group B patients. The number of ranibizumab injections per year in the 0.3 and 0.5 mg Group B patients was 4.19 and 4.27, respectively. The mean BCVA change (SD) from Month 12 to the last visit was )3.6 (14.82) letters for 0.3 mg (n = 28) and )2.2 (7.92) letters for 0.5 mg groups (n = 33) in Group B. Conjunctival haemorrhage and nasopharyngitis were the most commonly reported adverse events. Of the 13 serious adverse events reported, cerebral infarction (two incidences) was suspected to be study-drug related.

CONCLUSIONS: Pro re nata regimen of ranibizumab guided by monthly BCVA and other ophthalmic examinations appears effective in sustaining the BCVA gained with 12 monthly injections while reducing the number of injections during the extension phase. Ranibizumab was well tolerated during the extension phase.