Regulatory Pathway for a Sustained-Delivery Ophthalmic Platform

Challenge

The program combined a depot formulation with a delivery device, creating uncertainty on combination-product jurisdiction, nonclinical bridging, and clinical endpoints acceptable for both agencies. Internal teams had received mixed informal feedback; IND-enabling studies were at risk of rework. The board required a clear pre-IND and Scientific Advice plan before the next financing tranche.

Approach

We conducted a precedent-based gap analysis across FDA and EMA ophthalmic filings for sustained-release and intravitreal platforms, then built an interaction strategy with prioritized questions on CMC comparability, ocular pharmacokinetics, and durability endpoints. BEEÑA-E drafted briefing packages, facilitated mock agency Q&A, and aligned clinical and CMC leads on a single development narrative. Protocol refinements addressed injection burden, visit schedules, and imaging readouts before formal meetings.

Results

• Achieved productive pre-IND and EMA Scientific Advice outcomes with agreed Phase 2 endpoint framework
• Aligned CMC, device, and clinical teams on a single regulatory storyline and study sequence
• Initiated Phase 2 on the sponsor’s target timeline with protocol elements accepted by both agencies
• Avoided repeat nonclinical studies by clarifying bridging requirements in formal feedback
• Provided board-ready regulatory risk summary tied to capital plan milestones