Novartis said the FDA has accepted the company's Biologics License Application (BLA) for brolucizumab (RTH258) for the treatment of wet AMD. Brolucizumab is a humanized single-chain antibody fragment that is the most clinically advanced of its kind to reach this stage of development. Single-chain antibody fragments are highly sought after in drug development due to their small size, enhanced tissue penetration, rapid clearance from systemic circulation, and drug-delivery characteristics. The proprietary innovative structure results in a small molecule (26 kDa) with potent inhibition of, and high affinity to, all VEGF-A isoforms.
Seeking to make brolucizumab available as quickly as possible, Novartis used a priority review voucher to expedite FDA review in 6 months rather than the standard 10 months. If approved by the FDA, Novartis anticipates launching brolucizumab by the end of 2019. The regulatory application is primarily based on phase 3 data from the HAWK and HARRIER trials. The primary endpoint of these studies was noninferiority to aflibercept (Eylea; Regeneron) in mean change in BCVA from baseline to week 48. Novartis noted that HAWK and HARRIER are the first and only global head-to-head trials in patients with wet AMD that prospectively demonstrated efficacy at week 48 starting with a 12-week dosing regimen.