Roche Submits BLA for Port Delivery System with Ranibizumab for Wet AMD, Receives FDA Priority Review

On June 24, 2021, Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for the Port Delivery System with ranibizumab (PDS) for the treatment of patients with wet age-related macular degeneration (AMD). The FDA has also granted Priority Review to this BLA, with a target action date of October 23, 2021. A previous pivotal Phase 3 clinical trial demonstrated that in more than 98% of patients with wet AMD, PDS can extend the dosing interval to six months while providing visual outcomes comparable to monthly ranibizumab injections.

The press release stated that, if approved, PDS will become the first ocular implant therapy capable of periodic refills, providing a convenient new treatment option for patients with wet AMD as an alternative to the current standard of care.

The macula at the center of the retina is responsible for providing sharp central vision for activities such as reading. Wet age-related macular degeneration (wet AMD) is the leading cause of vision loss in individuals over the age of 60, affecting approximately 20 million patients worldwide. Wet AMD is an advanced ocular disease that can lead to rapid and severe vision loss. When abnormal blood vessels, known as "choroidal neovascularization (CNV)," grow into the macula, they leak fluid and blood, leading to scar tissue formation and ultimately damaging the central retina.

The current standard of care for this disease involves frequent intraocular injections of anti-vascular endothelial growth factor (VEGF) antibody drugs. However, patient responses to these medications vary, with some patients requiring more than 10 injections annually to achieve significant therapeutic efficacy. Furthermore, because these agents must be injected directly into the eye, frequent administration not only causes considerable inconvenience to patients' daily lives but also carries the risk of injection-related adverse effects.

The PDS enables the implantation of a refillable drug reservoir in the patient's eye, which is approximately the size of a grain of rice. It continuously delivers ranibizumab for up to several months, potentially reducing the treatment burden associated with frequent ocular injections and enhancing the durability of therapeutic efficacy. Ranibizumab is a vascular endothelial growth factor (VEGF) inhibitor that binds to and inhibits the VEGF-A protein, which plays a critical role in neovascularization and vascular leakage.

▲Introduction to the Port Delivery System (PDS) for Ranibizumab (Image source: Reference [2])

The submission of this Biologics License Application (BLA) is based on the positive results from the pivotal Phase 3 Archway clinical trial, a randomized, double-blind, multicenter, open-label study. The trial enrolled 418 patients with wet age-related macular degeneration (AMD) and was designed to evaluate the efficacy and safety of fixed-interval refills every 6 months using the Port Delivery System (PDS) compared with monthly intravitreal injections of 0.5 mg ranibizumab. The trial demonstrated that 98.4% (n=242/246) of patients in the treatment group did not require supplemental therapy during the first treatment interval.

Furthermore, the visual outcomes achieved in these patients were comparable to those in patients receiving monthly intravitreal ranibizumab injections. At Week 36, the mean change from baseline in central subfield thickness was within 10 μm in both groups. In terms of safety, the therapy was generally well tolerated in the trial, demonstrating a favorable benefit-risk profile.

References:

[1] FDA Accepts Application for Genentech’s Port Delivery System With Ranibizumab (PDS) for Treatment of Wet Age-Related Macular Degeneration (AMD). Retrieved June 24, 2021, from https://www.businesswire.com/news/home/20210623006007/en

[2] Roche Pharma Research and Early Development (pRED) Early Drug Development Investor Relations Event 2019. Retrieved November 11, 2019, from https://www.roche.com/dam/jcr:4b1ebb5e-e78d-4111-a380-1991422ad621/en/irp20191111.pdf

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