Genentech (Roche Group [SIX: RO, ROG; OTCQX: RHHBY]), has announced that the U.S. FDA has accepted a Biologics License Application (BLA), under priority review, for the company’s “Port Delivery System” with ranibizumab (PDS) for the treatment of wet, age-related macular degeneration (AMD). The PDS is a re-fillable eye implant, designed to continuously deliver a specific formulation of ranibizumab over a period of months. The strategy is aimed to reduce the treatment burden associated with frequent eye injections for its anti-VEGF treatment (ranibizumab). The submission is based on results from a Phase III study proposing that the PDS application may be able to use the ranibizumab treatment for up to six months.
The clinical trial (NCT03677934) was an RCT, multicenter, open-label Phase III study evaluating the efficacy and safety of the “Port Delivery System with ranibizumab (PDS)”, The PDS is an implantable, “reservoir-based, slow-release platform which sits in the sclera and delivers ranibizumab in a concentrated solution at different doses in the setting of nAMD”. According to the company, the PDS may be refilled every six months, compared to monthly intravitreal injections of ranibizumab 0.5 mg. In the two study arms (termed “ARCHWAY”), patients were treated with at least three anti-VEGF injections within the six months prior to their screening visit. The primary endpoint of the study was the change in BCVA score – including with correction such as glasses – from baseline at the average of week 36 and week 40. The study design used a non-inferior outcome, equivalent to monthly ranibizumab injections. The results reported by the company, indicated that, on average, patients had received five prior ranibizumab injections before their first study initiation visit. In the PDS arm of the study, patients gained an average of 0.2 eye chart letters in visual acuity from baseline compared with 0.5 eye chart letters for the monthly ranibizumab arm. During the first treatment interval, before the first scheduled refill, 1.6% of PDS patients assessed (n=4/246) received supplemental treatment, and 98.4% of patients (n=242/246) did not receive supplemental treatment.
Commenting on the application submitted to the FDA, Dr. Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development stated that, “anti-VEGF therapy brings significant benefit to people with wet AMD, 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. If approved, PDS would transform wet AMD 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.”