Dompé is one of only nine recipients of the new FDA voucher and the sole company in ophthalmology to be selected.. The CNPV shortens the standard marketing application review period from 10–12 months to just 1–2 months, significantly accelerating the potential path to approval.
NAION is the most common acute optic neuropathy in people over 50, typically causing sudden, irreversible vision loss due to ischemic damage to the optic nerve. Affecting approximately 6,000 Americans each year, its prevalence is rising alongside aging, diabetes, and cardiovascular disease — yet no approved therapies currently exist to halt or reverse its progression.
Dompé’s novel, intranasal NGF formulation (Cenegermin-bkbj) is designed to deliver the neurotrophic protein directly to the central nervous system, bypassing the blood–brain barrier. The approach builds on Dompé’s success with Oxervate® (cenegermin-bkbj)—the first FDA-approved NGF-based therapy, launched in 2018 for neurotrophic keratitis.
“The CNPV represents a strategic lever to accelerate access to transformative therapies for patients with unmet medical needs as NAION,” said Sergio Dompé, Executive President of the company. "Building on our pioneering achievement with Oxervate... we are now advancing a novel, intranasal formulation of NGF to bypass the blood-brain barrier and redefine possibilities in neuro-ophthalmology.”
Chief Development Officer Ahmed Enayetallah, added: “NAION, strongly associated with obesity, cardiovascular disease and an aging population, is the most common acute optic neuropathy in people over 50 and can lead to severe, permanent vision loss. Given this unmet need, we are thrilled by the opportunity to expedite its development and bring potential solutions to patients sooner through the CNPV voucher."
A global pivotal trial program is planned across 130 sites in 16 countries, evaluating intranasal NGF’s ability to preserve and restore visual function in NAION patients. The CNPV program — launched by the FDA in June 2025 — prioritizes breakthrough therapies addressing major US public health challenges.
Dompé’s selection highlights both the scientific maturity of NGF therapeutics and the growing recognition of neuro-ophthalmology as a frontier in regenerative medicine.
Source: Business Wire.
