FDA Selects Dompé for National Priority Voucher Program to Advance Intranasal NGF Therapy for NAION

Dompé has announced its selection by the U.S. Food and Drug Administration (FDA) to participate in the Commissioner’s National Priority Voucher (CNPV) program, an initiative designed to accelerate the development and review of innovative therapies addressing unmet medical needs.

Accelerated FDA Pathway for Intranasal Nerve Growth Factor Therapy

Through this designation, Dompé has been awarded a voucher granting a significantly shortened FDA review timeline for a future marketing application, as well as enhanced collaboration opportunities with the agency.

The company intends to apply this voucher toward the Biologic License Application (BLA) for its intranasal formulation of Nerve Growth Factor (Cenegermin-bkbj). This novel therapy is being developed for non-arteritic anterior ischemic optic neuropathy (NAION), a rare, vision-threatening condition currently without any approved treatments.

The intranasal NGF formulation represents a first-in-class, non-invasive delivery approach patented by Dompé. The therapy is designed to bypass the blood-brain barrier, targeting neural tissue more effectively and offering a potentially transformative approach to neuro-ophthalmic care.

“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.”

Understanding NAION: An Urgent Unmet Need

Non-arteritic anterior ischemic optic neuropathy (NAION) occurs when blood flow to the optic nerve is disrupted, leading to sudden and often irreversible vision loss. The condition affects approximately 6,000 new patients annually in the United States, a figure projected to increase as the population ages and as risk factors such as diabetes, hypertension, obesity, and sleep apnea become more prevalent.

As many as three out of four patients experience substantial visual impairment, and around 10% progress to legal blindness. Despite the considerable disease burden, no approved treatments currently exist to restore or preserve vision in NAION patients.

Global Clinical Development Plans

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