How Dompé Is Innovating in Neuro-Ophthalmology with the FDA’s CNPV Program

Vision loss driven by optic nerve injury remains one of ophthalmology’s toughest challenges. 

While treatment advancements have changed care for many retinal diseases, acute optic neuropathies, especially those linked to vascular and metabolic risk factors, still do not have effective, disease-modifying treatment options.

Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common acute optic neuropathy in adults over 50. NAION affects an estimated 6,000 people in the US each year and currently has no approved treatments. The condition involves sudden vision loss tied to optic nerve injury, which often becomes permanent. 

With aging populations and the rising prevalence of obesity, diabetes and cardiovascular disease, the clinical and societal impact of optic neuropathies is expected to grow.

Dompé, a privately held global biopharmaceutical company with deep roots in ophthalmology and neurology, is advancing a neuroregenerative science platform built around neurotrophins, including nerve growth factor (NGF). Neurotrophins are proteins that support neuronal growth, survival and repair. Among them, NGF is one of the most extensively studied, with decades of evidence supporting its relevance in neurodegenerative and neuro-ophthalmic disease.

Dompé is developing an intranasal NGF approach intended to preserve and improve visual function in patients with vision loss secondary to NAION, supported by plans for a large, global registrational trial program spanning more than 130 sites across 16 countries.

That work was recently highlighted by Dompé’s selection into the FDA’s Commissioner’s National Priority Voucher (CNPV) pilot program, an initiative designed to accelerate regulatory review for therapies targeting critical unmet medical needs.

To examine the scientific rationale, clinical challenges and regulatory significance behind this development, Xtalks spoke with Ahmed Enayetallah, MD, PhD, Chief Development Officer at Dompé. 

Enabling Bold Science in Areas of High Unmet Need

According to Dr. Enayetallah, Dompé’s ability to pursue high-risk, high-impact programs is tied to its organizational structure. With more than 130 years of independence as a privately owned company, Dompé combines long-term strategic stability with operational agility.

Unlike publicly traded biopharma companies, which often face short-term shareholder pressures, Dompé has greater flexibility to make patient-centric decisions and invest in areas of significant unmet need, even when those programs carry higher scientific or clinical risk.

This structural independence is closely linked with the company’s scientific foundation. Dompé’s research platform is deeply rooted in neurotrophin biology, particularly NGF, but also extends to additional neurotrophic factors that collectively support a broad and evolving pipeline. 

Together, the company’s governance model and scientific focus enable sustained investment in complex disease areas that others may be less equipped to pursue.

NGF Biology and Its Therapeutic Implications

NGF occupies a unique position in neuroscience, with more than 75 years of accumulated research spanning basic biology, translational studies and early clinical exploration. 

First identified in the 1950s, NGF’s biological significance was formally recognized in 1986, when Professor Rita Levi-Montalcini and Dr. Stanley Cohen were awarded the Nobel Prize in Physiology or Medicine for their discovery of neurotrophins and their role in nervous system development.

That long scientific history continues to inform modern therapeutic strategies. 

“NGF is a natural molecule, and its primary function is the survival of nerve cells, the maintenance of nerve cells, normal function and normal physiology of nerve cells and even reparative mechanisms for nerve cells,” said Dr. Enayetallah.

This wide range of biological activity is what makes NGF especially interesting in cases where there is neuronal injury, rather than just inflammation or degeneration.

In optic neuropathies, nerve fiber damage often causes permanent loss of function. Supporting neuronal survival while activating natural repair pathways offers a new approach to treatment.

Decades of preclinical models and human experience have shown NGF’s importance in neurological and eye diseases, including those that impact the optic nerve. 

However, as Dr. Enayetallah pointed out, the issue has not been a lack of biological reasoning. The real challenge is finding a way to deliver NGF effectively to deeply protected neural tissues. This issue has historically hindered its use in therapy. 

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Facing the Drug Delivery Challenge in Optic Nerve Disease

Drug delivery can be a big challenge, especially in neuro-ophthalmic disease. The optic nerve is physically isolated and protected by the blood-brain barrier and the blood-retina barrier. This severely limits how well systemically administered therapies can reach the affected tissues at levels that are beneficial.

“If you think of a condition that impacts the optic nerve, for example, an optic neuropathy, the optic nerve is buried so deep it’s almost like locked in a box, and it’s very hard to get to with systemic administration,” explained Dr. Enayetallah.

This challenge is particularly evident for protein-based molecules like NGF. Oral administration is not feasible due to rapid degradation in the GI tract. Parenteral routes, like intravenous or subcutaneous delivery, also provide limited access to CNS structures. Because of this, many biologics with promising neurobiology have had difficulty becoming effective treatments for optic nerve disorders.

Intranasal delivery is a different strategy. It uses direct pathways from the nose to the brain. This method can bypass both the blood-brain barrier and the blood-retina barrier. It offers a way to deliver therapeutic molecules more directly to the optic nerve and retinal ganglion cells.

Dompé’s intranasal platform is based on preclinical evidence supporting intranasal delivery. This approach allows long-studied neurotrophins like NGF to be tested in disease settings that were previously out of reach.

Measuring Meaningful Outcomes in Neuro-Ophthalmology Trials

In neuro-ophthalmology trials, demonstrating benefit requires outcomes that reflect how patients live their daily lives and how doctors evaluate disease progression. 

As Dr. Enayetallah noted, functional vision must be evaluated from more than one angle. “You want to look at things like best-corrected visual acuity… [but] it’s really important to look at both visual acuity, the sharpness, but also the visual field, how much of the world patients can see.” Loss of peripheral vision, even if central acuity remains intact, can still qualify as legal blindness and greatly restrict independence. 

To support these functional measures, structural endpoints clarify whether a treatment is affecting the underlying neural issue. Imaging tools like optical coherence tomography (OCT) let researchers examine retinal layers and optic nerve fibers in detail, revealing information about neuronal health. When structural findings can be aligned with changes in visual acuity or visual field, they offer clinicians and researchers a clearer understanding of therapeutic effect.

This combination of functional and structural endpoints allows for a more thorough assessment of neuroprotective and reparative approaches in optic nerve disease.

Regulatory Innovation and the Impact of the FDA’s CNPV Program

Dompé’s selection into the FDA’s CNPV program represents formal recognition of both the severity of NAION and the absence of available therapies. 

Among just nine programs chosen in the pilot’s first round, Dompé was the only company advancing an ophthalmic indication.

As Dr. Enayetallah explained, the designation reflects more than regulatory speed; it signals prioritization of an area long defined by limited options.

“It’s almost some sort of external validation of the unmet need and the seriousness of the condition,” he said.

 The voucher allows for a significantly shorter review timeline, reducing the typical 10 to 12 months to only one or two months. It also promotes closer and more ongoing communication with FDA review teams. 

For patients facing vision loss, which is often sudden and irreversible, this faster process has real-world effects. Early regulatory review may lead to quicker access to potential treatments in a condition where lost time often means lost function. 

Targeting Root Causes in Optic Neuropathy

Rather than focusing solely on managing downstream consequences of vision loss, Dr. Enayetallah frames Dompé’s approach as an effort to intervene at the level of underlying pathology. 

In conditions associated with aging and cardiometabolic disease, he noted that current care often addresses symptoms without altering the disease course. By contrast, neurotrophin-based approaches aim to preserve or restore physiological function at the site of neural injury.

“What we’re trying to do here in NAION is really a potentially disease-modifying treatment. We are trying to either stop or reverse the pathology and restore the physiological function,” he noted. This focus on treating the root cause marks a significant change in how optic neuropathies are treated. 

In addition to developing treatments, the program also raises awareness about a condition that has often been overlooked. 

Dr. Enayetallah pointed to the lack of visibility and advocacy in NAION compared with other diseases, adding that “by doing the work, in a way, we are almost a public health advocate for this disease area.” 

Through research and community engagement, these efforts can help improve the understanding and recognition of NAION in both clinical and public health circles.