Abstract
Purpose :
No pharmacological therapy is approved for the treatment of childhood myopia progression in the U.S. and Europe. This double-masked, placebo-controlled, parallel-group, randomized phase 3 study (NCT03350620) assessed the safety and efficacy of NVK002, a novel, preservative-free, low-dose atropine (LDA) formulation, in slowing myopia progression after 3 years of treatment.
Methods :
US and European participants aged 3 to 17 years with −0.50 D to −6.00 D spherical equivalent refraction (SER) and no worse than −1.50 D astigmatism were randomized in 2:2:3 ratio to receive once-daily placebo, NVK002 0.01%, or 0.02% eye drop for 36 months. The primary and secondary efficacy endpoints were the proportion of responders (<0.50 D myopia progression over 36 months) and mean change from baseline in SER or axial length at Month 36 in a modified intent-to-treat (mITT) population (participants 6 to 10 years old at baseline).
Results :
In total, 576 patients were randomized: 573 received study treatment and 489 were in mITT. Both doses slowed myopia progression. At Month 36, compared with placebo, NVK002 0.01% significantly increased the proportion of responders (p=0.031) and significantly slowed mean SER progression (p<0.001) and axial elongation (p<0.001); NVK002 0.02% significantly slowed mean axial elongation (p=0.005; Months 12-36) but did not significantly increase the proportion of responders (p=0.373) nor slow mean SER progression (p=0.101; except Month 12). No serious ocular treatment-emergent adverse events (TEAE) occurred. Serious non-ocular TEAEs were reported in 2.3% of patients (placebo, 2.5%; NVK002 0.01%, 0.6%; 0.02%, 3.2%) and were not considered drug related. The incidence of any TEAE was similar across treatments, most commonly photophobia (4.5%), allergic conjunctivitis (3.3%), eye irritation (1.6%), and mydriasis (1.0%).
Conclusions :
For the first time in a U.S. and European sample, it has been demonstrated that LDA can have a meaningful impact on myopia progression. NVK002 0.01% demonstrated significant efficacy across all three main efficacy endpoints compared to placebo over 3 years, while both NVK002 concentrations had an excellent safety profile. NVK002 0.01% may offer the first approved pharmacological treatment option for myopia progression.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.