Novartis has recieved FDA filing acceptance and Priority Review of brolucizumab (RTH258) for patients with wet AMD.
Novartis has announced that the US Food and Drug Administration (FDA) accepted the company’s Biologics License Application (BLA) for brolucizumab (RTH258) for the treatment of wet age-related macular degeneration (AMD), also known as neovascular AMD, or nAMD.
Seeking to make brolucizumab available as quickly as possible, Novartis used a priority review voucher to expedite FDA review. If the Priority Review of brolucizumab is approved by the FDA, Novartis anticipates launching by the end of 2019.
nAMD (neovascular age-related macular degeneration or wet AMD) is the leading cause of severe vision loss and legal blindness in people over the age of 65 in North America, Europe, Australia and Asia. nAMD occurs when abnormal blood vessels form underneath the macula, the area of the retina responsible for sharp, central vision. These blood vessels are fragile and leak fluid, disrupting the normal retinal architecture and ultimately causing damage to the macula.
Early symptoms of nAMD include distorted vision or metamorphopsia and difficulties seeing objects clearly. Prompt diagnosis and intervention are essential. As the disease progresses, cell damage increases, further reducing vision quality. This progression can lead to a complete loss of central vision, leaving the patient unable to read, drive or recognise familiar faces. Without treatment, vision can rapidly deteriorate.
“Wet AMD robs people of their precious sight and takes a major toll on the lives of millions of people”
Estimates suggest that by 2020, 1.5 to 1.75 million people in the US will be living with wet AMD, a leading cause of blindness worldwide and a rapidly growing public health concern. As the disease progresses, patients may experience loss of central vision, resulting in an inability to complete daily tasks. Without treatment, vision can rapidly deteriorate and may lead to blindness.
The regulatory application is primarily based on Phase III data from the HAWK and HARRIER trials – prospective, randomised, double-masked multi-center studies. The primary endpoint of these studies was non-inferiority to aflibercept in mean change in best-corrected visual acuity (BCVA) from baseline to week 48 (mean change in BCVA of 6.6 letters for brolucizumab 6 mg versus 6.8 letters for aflibercept in HAWK and 6.9 letters versus 7.6 letters, respectively, in HARRIER). 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.
Additionally, at week 48 in the studies, key secondary endpoint assessments showed significantly fewer brolucizumab patients with disease activity (23.5% of brolucizumab 6 mg patients versus 33.5% of aflibercept patients in HAWK, and 21.9% versus 31.4%, respectively, in HARRIER as well as retinal fluid – key markers used by physicians to help guide management of the disease in clinical practice (31% fewer patients on brolucizumab 6 mg had intra-retinal fluid (IRF) and/or sub-retinal fluid (SRF) in HAWK, and 26% fewer in HARRIER, versus aflibercept.
Commenting on the application for Priority Review of brolucizumab, Fabrice Chouraqui, President, Novartis Pharmaceuticals Corporation said, “Reaching this milestone is an important step in our efforts to re-imagine the treatment journey for people with wet AMD and their caregivers. We are looking forward to the potential of a new option for patients with wet AMD, who often have to navigate considerable physical and emotional difficulties caused by deteriorating vision.”
Dawn Prall George, executive director, The Support Sight Foundation said, “Wet AMD robs people of their precious sight and takes a major toll on the lives of millions of people who face not only vision loss, but also the burden of frequent injections into their eyes. We are always excited about potential new treatment options and hopeful they may help people manage this devastating disease.”