FDA accepts application for Roche’s Port Delivery System with ranibizumab (PDS) for treatment of neovascular or “wet” age-related macular degeneration (nAMD)

Roche announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA), under Priority Review, for Port Delivery System with ranibizumab (PDS) for the treatment of neovascular or “wet” age-related macular degeneration (nAMD). Neovascular AMD is a leading cause of blindness for people aged 60 and over and impacts approximately 20 million people worldwide. If approved, PDS would be a first-of-its-kind therapeutic approach, offering people living with nAMD an alternative to frequent eye injections of anti-vascular endothelial growth factor (VEGF), the current standard of care. The FDA is expected to make a decision on approval by 23 October 2021.

“Anti-VEGF therapy brings significant benefit to people with nAMD, but optimal results require frequent trips to the doctor’s office for eye injections. This burden leaves many people under-treated and susceptible to vision loss,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “If approved, PDS would transform nAMD treatment by providing up to six months of uninterrupted therapy that could potentially improve vision outcomes compared to what is currently achieved in the clinic.”

PDS is a permanent refillable eye implant, approximately the size of a grain of rice, designed to continuously deliver a customised formulation of ranibizumab over a period of months, potentially reducing the treatment burden associated with frequent eye injections.

The BLA submission is based on positive results from the phase III Archway study primary analysis, which showed that of those nAMD patients being treated with PDS, more than 98% were able to go six months without needing additional treatment prior to the refill-exchange. In addition, these patients achieved vision outcomes equivalent to patients receiving monthly ranibizumab eye injections. In the study, PDS was generally well-tolerated, with a favourable benefit-risk profile. The safety profile of PDS in the clinical trial setting is well understood and will continue to be closely monitored. If approved, PDS would be the first and only nAMD therapy indicated to allow six months between treatments.

Roche has a robust phase III clinical development programme underway for PDS, including the Portal, Pagoda and Pavilion studies. Portal is an extension study evaluating the long-term safety and efficacy of PDS in nAMD. Pagoda is evaluating PDS for the treatment of diabetic macular edema (DME), while Pavilion is a study of PDS in diabetic retinopathy without DME. Both the Pagoda and Pavilion trials are actively recruiting participants.

The PDS Marketing Authorisation Application has also been validated by the European Medicines Agency and is currently under review.