Dompé's Program in NAION Selected for Landmark FDA Commissioner’s National Priority Voucher Program

Dompé, a leading biopharmaceutical company with operations in Italy and the US, announced today its selection by the United States Food and Drug Administration (FDA) to participate in the Commissioner’s National Priority Voucher (CNPV) program. Because of its selection, Dompé has been awarded a voucher granting a significantly shortened review timeline for marketing application and enhanced collaboration with the FDA. Dompé intends to apply the voucher for the Biologic License Application (BLA) for an intranasally administered formulation of Nerve Growth Factor (Cenegermin-bkbj) for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION). The intranasal administration of NGF is a novel approach, patented by Dompé, offering a non-invasive delivery method.

“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 at 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 bypass the blood-brain barrier and redefine possibilities in neuro-ophthalmology.”

NAION refers to a loss of blood flow to the optic nerve which causes sudden vision loss, that usually occurs spontaneously. There are approximately 6,000 new cases in the United States per year, and the number of cases is growing because of the aging population and rising prevalence of risk factors such as diabetes, hypertension, and other obesity-related conditions like sleep apnea. As many as three out of four patients experience substantial visual impairment, and approximately one in 10 patients experience profound loss of vision and legal blindness.¹

There are currently no approved treatments to reverse vision loss or prevent worsening in patients with NAION.

“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. “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."

Dompé is developing intranasal NGF with aims of preserving and improving visual function in patients with vision loss secondary to NAION. A registrational pivotal trial program is planned in more than 130 sites across 16 countries.

About Dompé
Dompé farmaceutici S.p.A. is a privately held, global biopharmaceutical company on a mission to bring the full potential of nerve growth factor (NGF) to patients. As the first company to unlock the therapeutic potential of NGF, Dompé developed the first FDA-approved NGF treatment. Today, our clinical pipeline reflects our commitment to redefining treatments across ophthalmic, neurological and pain-related conditions. Building on 130 years of independence, we are embracing the challenge to transform our science and ourselves by delivering first-in-class, disease-modifying therapies through NGF and other breakthrough molecules to help people improve their sight, support neurological recovery and manage pain effectively. Today, Dompé employs more than 950 employees worldwide and maintains a US commercial operations hub in the San Francisco Bay Area.

Learn more at https://www.dompe.com/us/.

About Nerve Growth Factor (NGF)
Nerve Growth Factor (NGF) is a neurotrophin, a member of a family of proteins essential for the growth, maintenance and survival of neurons, with broad therapeutic potential. NGF was discovered by the Italian neurobiologist Professor Rita Levi-Montalcini and American biochemist Dr. Stanley Cohen, earning them the Nobel Prize in Physiology or Medicine in 1986. In their research, Drs. Levi-Montalcini and Cohen uncovered that NGF plays a crucial role in the proliferation, differentiation and survival of sympathetic and sensory neurons. Dompé is the first company to unlock the therapeutic potential of NGF, building upon a Nobel Prize-winning discovery to deliver a breakthrough treatment for a rare neurotrophic eye disease.​

About Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)
Optic neuropathies are disorders caused by damage to the optic nerve, disrupting visual signals from the retina to the brain due to injury, inflammation, poor blood flow, or genetic and degenerative factors. The result is progressive or sudden loss of vision, often affecting central or peripheral sight, color discrimination, and in severe cases, leading to permanent blindness. Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common cause of acute optic nerve injury and optic neuropathy in people over 50. It affects men and women equally, usually starting around age 66. While the exact cause isn’t fully understood, common risk factors include diabetes, high blood pressure, high cholesterol, and smoking. Despite its frequency, and risk of severe permanent vision loss, there is no proven treatment to improve visual outcomes.

About the Commissioner's National Priority Voucher (CNPV) Pilot Program
The U.S. Food and Drug Administration (FDA) launched the Commissioner’s National Priority Voucher (CNPV) pilot program to accelerate the development and review of prescription drugs and biological products that address critical U.S. national health priorities. Announced in June 2025, the program offers companies the opportunity to reduce standard application review times from 10–12 months to just 1–2 months through a collaborative, multidisciplinary review process.

Selected companies receive a voucher that provides enhanced communication with the FDA and a rolling review to facilitate a faster timeline, while maintaining the agency’s rigorous standards for safety and efficacy. The program focuses on transformative therapies that tackle major public health challenges and large unmet medical needs.

About Oxervate
OXERVATE^® (cenegermin-bkbj) ophthalmic solution 0.002% is indicated for the treatment of neurotrophic keratitis. Important Safety Information: Contact lenses, either therapeutic or corrective, should be removed before applying OXERVATE. Contact lenses may be reinserted 15 minutes after OXERVATE administration. Eye Discomfort, such as eye pain, that can be mild to moderate can occur with OXERVATE. Patients should contact their health care provider if a more serious eye reaction occurs. The most common adverse reaction with OXERVATE (~16%) was eye pain. Other adverse reactions with OXERVATE (1% to 10%) included corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation, photophobia, tearing, and headache.

References:

1. Dattilo M, et al. Neuroophthalmology. 2020;45(1):23-28.

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