How Dompé Is Innovating in Neuro-Ophthalmology with the FDA’s CNPV Program

Key Takeaways

• NAION affects ~6,000 US patients annually, no approved therapy
• Dompé uses intranasal NGF to target optic nerve repair
• FDA CNPV selection accelerates review to 1‑2 months
• Intranasal delivery bypasses blood‑brain and blood‑retina barriers
• Trial across 130 sites, 16 countries, functional and structural endpoints

Summary

Dompe, a privately owned biopharma, is advancing an intranasal nerve growth factor (NGF) therapy to treat non‑arteritic anterior ischemic optic neuropathy (NAION), a leading cause of sudden vision loss affecting roughly 6,000 U.S. patients annually. The approach leverages NGF’s neuroprotective properties and a novel nasal‑to‑brain delivery system to bypass the blood‑brain and blood‑retina barriers. Dompe’s program was selected for the FDA Commissioner’s National Priority Voucher (CNPV), which can compress the regulatory review timeline to one‑to‑two months. A global registrational trial will span more than 130 sites across 16 countries, measuring both functional vision and structural retinal outcomes.

Pulse Analysis

Non‑arteritic anterior ischemic optic neuropathy (NAION) is the leading acute optic nerve disorder in adults over 50, causing sudden, often permanent vision loss. With an estimated 6,000 new cases each year in the United States and a growing burden from obesity, diabetes and cardiovascular disease, the condition represents a rising public‑health challenge. Despite advances in retinal therapeutics, NAION remains without disease‑modifying options, leaving clinicians to manage only the symptoms. The lack of approved treatments underscores a critical gap that biopharma companies are eager to fill.

Dompe’s strategy builds on a decades‑long neurotrophin platform centered on nerve growth factor (NGF), a protein known to support neuronal survival and repair. Recognizing that systemic delivery cannot reach the protected optic nerve, the company has pioneered an intranasal formulation that exploits direct nose‑to‑brain pathways, effectively bypassing both the blood‑brain and blood‑retina barriers. Preclinical data demonstrate that intranasal NGF can reach retinal ganglion cells and promote axonal regeneration, offering a mechanistic route to preserve visual function. This delivery innovation transforms a biologically compelling molecule into a viable therapeutic candidate for NAION.

The FDA’s Commissioner’s National Priority Voucher (CNPV) program has placed Dompe’s NAION program among nine elite candidates, granting a review window of one to two months instead of the usual ten‑plus months. This regulatory acceleration not only shortens time to market but also deepens interaction with FDA reviewers, potentially de‑risking the large global registrational trial that will involve over 130 sites in 16 countries. Success could set a precedent for fast‑tracking neuro‑ophthalmic therapies, encouraging other innovators to pursue high‑risk, high‑impact projects in underserved disease areas.