Roche Submits BLA for Ranibizumab Port Delivery System (PDS) with Twice-Yearly Refills for Wet AMD
Jun 27, 2021 02:04
CST Updated
02:04
Genentech
Pharmaceutical R&D Manufacturer
Roche
Oncology Drug Research, Development, and Manufacturing
FDA
U.S. Food and Drug Administration
nAMD (Image source: medscape.com)
June 26, 2021 /BioonBIOON/ -- Genentech, a member of the Roche Group, recently announced that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) submitted by the company, which is currently under priority review. The BLA pertains to a novel intraocular drug delivery system, ranibizumab PDS (ranibizumab intravitreal implant), for the treatment of neovascular age-related macular degeneration (nAMD, also known as "wet AMD").FDAAn approval decision is expected by October 23, 2021. In Europe, the Marketing Authorization Application (MAA) for PDS has been accepted by the European Medicines Agency (EMA) and is currently under review.
The Port Delivery System (PDS) is a permanent, refillable intraocular implant approximately the size of a grain of rice that continuously delivers a customized formulation of ranibizumab over several months, with the potential to reduce the treatment burden associated with frequent intravitreal injections.
The PDS allows for continuous refill (once every 6 months). If approved, the PDS will become the first and only intraocular implant for sustained drug delivery, offering patients with wet AMD an alternative to frequent intravitreal anti-vascular endothelial growth factor (VEGF) injections, which are currently the standard of care for wet AMD.
Dr. Levi Garraway, Chief Medical Officer and Global Head of Product Development at Roche, stated: "Anti-VEGF therapies offer significant benefits to patients with wet AMD, but achieving optimal outcomes requires patients to frequently visit hospitals for intravitreal injections. This burden leaves many patients undertreated and susceptible to vision loss. If approved, the PDS could transform the treatment of wet AMD by providing continuous therapy for up to six months, potentially improving vision compared to current clinical outcomes."
PDS (Image source: PMID: 30946888)The PDS BLA submission is based on the positive results from the Phase 3 ARCHWAY study (NCT03677934). The study met its primary endpoint, with data demonstrating that more than 98% of patients with wet AMD treated with the PDS did not require additional treatment for up to 6 months prior to refill. Furthermore, the visual acuity outcomes achieved by these patients were consistent with those of patients receiving monthly 0.5 mg ranibizumab injections. In this study, the PDS was well tolerated and demonstrated a favorable benefit-risk profile.
PDS inClinical TrialThe safety profile has been well characterized and will continue to be closely monitored. If approved, PDS will be the first and only wet AMD therapy that allows for a 6-month dosing interval.
PDS Refill Procedure Schematic (retinalphysician.com)
Neovascular age-related macular degeneration (nAMD) is the leading cause of blindness globally among individuals aged 60 years and older. The current standard of care for nAMD requires patients to visit an ophthalmologist monthly to receive intravitreal anti-vascular endothelial growth factor (anti-VEGF) injections to help maintain visual gains and/or prevent vision loss. The high treatment burden associated with anti-VEGF therapy may lead to undertreatment of nAMD and potentially suboptimal visual outcomes.
Ranibizumab is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to and inhibit VEGF-A, a protein that plays a key role in neovascularization and vascular leakage.
The PDS contains a customized ranibizumab formulation that has not been approved by regulatory authorities. It differs from ranibizumab intravitreal injection (Lucentis®, ranibizumab injection), which has been approved for the treatment of nAMD and other retinal diseases.
By maintaining therapeutic ranibizumab concentrations through twice-yearly refills, the PDS can provide nAMD patients with greater certainty of outcomes in achieving and sustaining visual acuity gains. Additionally, by reducing the need for frequent injections and physician visits, the PDS can alleviate the treatment burden associated with standard anti-VEGF therapy.
In addition to the ARCHWAY study, the PORTAL study is investigating the long-term safety and tolerability of PDS for the treatment of nAMD. Additionally, the PAGODA study is investigating PDS for the treatment ofDiabetes mellitusMacular edema (DME), which isDiabetesvision-threatening complications. (Bioon.com)