Dompé Announces FDA-Approved First-in-Class Therapy Oxervate for Neurotrophic Keratitis Following Industry Innovation Award

VCBeat (WeChat ID: vcbeat) has learned that the U.S. National Organization for Rare Disorders (NORD) recently announced it had awarded Dompé, a biopharmaceutical company, with the 2019 Industry Innovation Award. The company’s flagship product, Oxervate eye drops, used to treat neurotrophic keratitis (NK), received approval from the U.S. Food and Drug Administration (FDA) in August 2018, marking the first FDA-approved therapy for NK.

Dompé is a biopharmaceutical company headquartered in Milan, Italy, dedicated to developing innovative solutions for various diseases with high patient populations and a lack of standard treatment options. Its research areas include diabetes, organ transplantation, ophthalmic diseases, and oncology. The company employs 700 people worldwide and has offices in Albania, France, Germany, the United Kingdom, Spain, and the United States (New York).

The cornea is the most densely innervated tissue in the human body, with approximately 7,000 nerve endings per square millimeter. Corneal nerves maintain ocular surface health by producing soluble factors known as neurotrophic factors, in conjunction with the blink and tear reflexes.

Neurotrophic keratitis is a degenerative disease caused by loss of corneal sensation, associated with damage to the trigeminal nerve (one of the nerves responsible for ocular anatomy and function). Such damage can lead to loss of corneal sensitivity and, in severe cases, result in corneal thinning, ulceration, and perforation, thereby impairing patients’ vision. Although the incidence of neurotrophic keratitis is low, affecting fewer than 5 individuals per 10,000 people, its impact on patients is devastating; surgical intervention is often required, and postoperative risks remain substantial.

Oxervate is a topical solution for the treatment of neurotrophic keratitis, administered six times daily at two-hour intervals for eight weeks. Studies have shown that 72% of patients achieved complete corneal healing within eight weeks, compared to only 28% of patients not treated with Oxervate eye drops.

Oxervate’s primary active pharmaceutical ingredient, cenegermin, is a recombinant form of human nerve growth factor (NGF), discovered by Nobel laureate Rita Levi-Montalcini. This protein is naturally produced in the human body and plays a role in the development, maintenance, and survival of nerve cells.

Sergio Dompé, Chairman of Dompé, stated: “By leveraging recombinant DNA technology, we introduced a specific gene into bacteria to enable the production of human nerve growth factor (NGF), thereby facilitating the repair of corneal damage. This approach marks the first successful translation of nerve growth factor into a therapeutic agent, helping patients reduce surgery-related risks. Neurotrophic keratitis (NK) can be cured by adhering to the prescribed dosage and schedule of Oxervate eye drops.”

Peter L. Saltonstall, President and CEO of NORD, also stated, “In the United States, 30 million people are affected by rare diseases. We have been committed to seeking treatments for rare diseases. We thank Dompé for its contributions to patients with rare diseases and their caregivers, and we hope that Dompé will continue to provide more breakthrough therapies in the field of orphan diseases after receiving the Industry Innovation Award.”

(Compiled by Feng Yutong)