An Italian-based pharmaceutical company with subsidiaries in New York has been granted orphan drug status for a new drug to treat neurotrophic keratitis, a rare degenerative corneal disease that impacts less than 1 in 5,000 people worldwide.
It can be caused by several different existing conditions, including diabetes, and there are currently no curative treatments available.
The drug – based on recombinant human nerve growth factor, or rhNGF – was developed in Italy at L’Aquila laboratories of the Biopharmaceutical Group, founded on the research of Nobel Prize winner Rita Levi Montalcini, who isolated nerve growth factor in 1952 and continued her research for decades, earning the Nobel Prize in 1986.
Recombinant human nerve growth factor, produced by transferring human genetic material into bacterial DNA, works by enabling the bacteria to produce nerve growth factor that is identical to that naturally produced by the human body, helping to stimulate the growth and maintenance of nerve cells. The cornea is particularly dense with nerves, making it an ideal site for an innovative treatment option.
The FDA and the European Medicines Agency had already assigned rhNGF orphan drug status for the treatment of retinitis pigmentosa, another eye disease without cure that impacts more than one million people worldwide.
The drug is currently in the advanced stages of a double-blind study based in nine European countries involving the treatment of patients suffering from neurotrophic keratitis. More than 170 patients are enrolled in the study, which is designed to evaluate corneal lesion healing, improvement in visual acuity and sensitivity of the cornea.
“We are particularly proud of this new FDA designation, which represents an important recognition of our Research & Development efforts in the field of ophthalmology on a global scale, particularly in North America,” said Eugenio Aringhieri, CEO of the Dompé Group, which focuses on the treatment of rare diseases related to diabetes, organ transplants and oncology. “It is another confirmation of the constant dialogue with the international regulatory agencies in order to bring health issues that still need to be resolved to the forefront as well as possible therapeutic solutions for diseases that are currently without a cure. This is the direction that the efforts of our group is taking, focused on the identification of innovative drugs to protect patients around the world.”
Recumbent human nerve growth factor has also been used to treat peripheral neuropathy.