FDA Selects Dompé for National Priority Voucher Program to Accelerate Development of Novel Intranasal Therapy

Dompé announced its selection by the FDA to participate in the Commissioner’s National Priority Voucher (CNPV) program.

Through this designation, Dompé has been awarded a voucher granting a significantly shortened FDA review timeline for a future marketing application, along with enhanced collaboration opportunities with the agency. The company said it intends to apply this voucher toward the Biologic License Application (BLA) for its intranasally administered formulation of Nerve Growth Factor (Cenegermin-bkbj), developed for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION)—a rare, vision-threatening condition with no approved therapies.

The intranasal formulation of NGF represents a first-in-class, noninvasive delivery approach, patented by Dompé, designed to bypass the blood-brain barrier and target neural tissue more effectively.

“The CNPV represents a strategic lever to accelerate access to transformative therapies for patients with unmet medical needs such as NAION,” said Sergio Dompé, Executive President of Dompé. “Building on our pioneering achievement with Oxervate—the first NGF-based therapy approved for neurotrophic keratitis in 2018—we are now advancing a novel, intranasal formulation of NGF (Cenegermin-bkbj) to redefine possibilities in neuro-ophthalmology.”

NAION occurs when blood flow to the optic nerve is disrupted, leading to sudden, often irreversible vision loss. It affects approximately 6,000 new patients in the United States each year, a number expected to rise with the aging population and increasing prevalence of risk factors such as diabetes, hypertension, obesity, and sleep apnea. As many as three out of four patients experience substantial visual impairment, and roughly 10% suffer profound vision loss leading to legal blindness. Despite the significant disease burden, there are currently no approved treatments to restore or preserve vision in NAION patients.

Dompé is advancing intranasal NGF as a potential therapy to preserve and improve visual function in patients with NAION-related vision loss. The company plans a registrational, pivotal clinical trial program spanning more than 130 sites across 16 countries.