Dompé Receives FDA Priority Voucher for Intranasal NGF Therapy Targeting NAION Vision Loss
核心洞察
Dompé (搜索) has been selected as one of only nine recipients for the FDA's Commissioner's National Priority Voucher program, receiving the sole award in ophthalmology.
The voucher will accelerate review of the company's Biologics License Application for intranasal Cenegermin-bkbj, reducing standard review time from 10-12 months to just 1-2 months.
The therapy targets non-arteritic anterior ischemic optic neuropathy (NAION), which affects approximately 6,000 new patients annually in the US with no current approved treatments.
Dompé (搜索), a leading biopharmaceutical company, has been selected by the FDA to participate in the Commissioner's National Priority Voucher (CNPV) program, making it one of just nine voucher recipients and the only company chosen in the ophthalmology space. The selection grants Dompé a voucher that significantly shortens the marketing application review timeline from the standard 10-12 months to just 1-2 months.
The company plans to apply the voucher to its Biologics License Application (BLA) for an intranasal formulation of Nerve Growth Factor (Cenegermin-bkbj) for treating non-arteritic anterior ischemic optic neuropathy (NAION). This novel intranasal administration approach, patented by Dompé (搜索), offers a non-invasive delivery method designed to bypass the blood-brain barrier.
Addressing Critical Unmet Medical Need
NAION represents a significant unmet medical need, affecting approximately 6,000 new patients annually in the United States. The condition involves loss of blood flow to the optic nerve, causing sudden vision loss that typically occurs spontaneously. The incidence is growing due to an aging population and rising prevalence of risk factors including diabetes, hypertension, and obesity-related conditions such as sleep apnea.
The disease burden is substantial, with as many as three out of four patients experiencing substantial visual impairment, and approximately one in 10 patients suffering profound vision loss and legal blindness. Currently, no approved treatments exist to reverse vision loss or prevent worsening in NAION patients.
"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," said Ahmed Enayetallah, Chief Development Officer at Dompé (搜索). "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."
Building on Nobel Prize-Winning Science
Dompé (搜索)'s intranasal NGF platform builds upon the Nobel Prize-winning discovery of neurobiologist Professor Rita Levi-Montalcini and biochemist Dr. Stanley Cohen, who earned the Nobel Prize in Physiology or Medicine in 1986 for their work on Nerve Growth Factor. Their research revealed that NGF plays a crucial role in the proliferation, differentiation, and survival of sympathetic and sensory neurons.
The company has previously demonstrated success in translating NGF science into approved therapies, having developed Oxervate—the first NGF-based therapy approved for Neurotrophic Keratitis in 2018.
"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 bypass the blood-brain barrier and redefine possibilities in neuro-ophthalmology," said Sergio Dompé (搜索), Executive President at Dompé.
Clinical Development Program
Dompé (搜索) is developing the intranasal NGF therapy with the aim of preserving and improving visual function in patients with vision loss secondary to NAION. The company has planned a registrational pivotal trial program spanning more than 130 sites across 16 countries.
The FDA's CNPV pilot program, announced in June 2025, was designed to accelerate development and review of prescription drugs and biological products addressing critical U.S. national health priorities. Selected companies receive enhanced communication with the FDA and a rolling review process while maintaining rigorous safety and efficacy standards.
Company Background
Dompé (搜索) farmaceutici S.p.A. is a privately held, global biopharmaceutical company focused on bringing the full potential of nerve growth factor to patients. As the first company to unlock NGF's therapeutic potential, Dompé has built a clinical pipeline targeting ophthalmic, neurological, and pain-related conditions. The company employs more than 950 people worldwide and maintains U.S. commercial operations in the San Francisco Bay Area, building on 130 years of independence in pharmaceutical development.