Cenegermin Eye Drops Receive European Union Approval: The First Biotechnological Drug Resulting from

Behind the development of cenegermin are decades of Italian research, stemming from the studies of Rita Levi Montalcini, winner of the Nobel Prize in Medicine for the discovery of the nerve growth factor (NGF).  

In 2015, the product was designated as an orphan drug in Europe for the treatment of neurotrophic keratitis, a rare eye disease that affects less than 5 out of every 10,000 people[1]. Until now, there have been no approved drugs for this condition. 

Dompé, an Italian biopharmaceutical company, announces that the European Commission has granted the marketing authorisation of cenegermin eye drops for the treatment of moderate to severe neurotrophic keratitis. This makes it the first biotechnological treatment authorized for this specific indication in the Europe Union, which responds to the needs of patients affected by this rare and severely debilitating eye disease that can cause vision loss. Until now, there have been no approved drugs for the treatment of this condition.

Behind the development of cenegermin are decades of "Made in Italy" research, stemming from the studies of Nobel laureate Rita Levi Montalcini, who discovered the nerve growth factor (NGF). This lead to the collaboration between Dompé and world-renowned centres of excellence in ophthalmology.

"Neurotrophic keratitis is a rare disease that is particularly debilitating, and until now, has been an unresolved health concern for patients. The authorisation obtained by the European Commission, after positive opinion received from the EMA CHMP, is a milestone for the patients affected by this disease, for the research in this sector and for our Company," affirms Eugenio Aringhieri, Chief Executive Officer of Dompé Pharmaceuticals. "It is our first biotech drug that was obtained thanks to the work of a skilled and passionate team that has believed in this project from the beginning; and thanks to the constant collaboration with an excellent medical community, which has contributed to the development of the product in its various phases in national and international centres. It is a long, complex yet exciting journey that never ends but begins with this important regulatory step. The next steps will definitely be to make the product available in other areas outside of Europe and in other future indications."

Cenegermin is the name of the primary active ingredient of the drug, and it is the recombinant version of the human nerve growth factor (NGF), developed and prepared through a biotechnological production process unique to Dompé. Cenegermin is a protein that is almost identical to the one the human body produces naturally, which is involved in the development, maintenance and survival of nerve cells[2]. Administered as eye drops in patients with moderate to severe neurotrophic keratitis, this solution can help restore the normal healing processes of the eye and repair corneal damage.

"There are numerous clinical conditions that can lead to subnormal corneal sensitivity and neurotrophic keratitis due to damage to the rich nerve supply provided to the eye via the trigeminal nerve", says Reza Dana, Professor of Ophthalmology at Harvard Medical School, and Director of the Cornea Service and Senior Scientist at the Massachusetts Eye and Ear, Boston, USA. "In the more severe cases, neurotrophic keratitis can lead to ulcers, corneal necrosis and perforation, and visual loss. Considering the possible severity of neurotrophic keratitis, and the lack of good response to standard therapies in many cases, having a valid therapeutic option that can directly promote nerve function is a major contribution to the field and to the community of ophthalmologists, not to mention the many patients suffering from this condition."

Cenegermin was developed at the Dompé L'Aquila Research and Production Centre, thanks to recombinant DNA technology, with the use of bacteria in which a gene (DNA) is introduced that allows the bacteria to produce the human growth factor on their own.

"I think it is a big success to have taken the first nerve growth factor from Rita Levi Montalcini's discovery after so many years of hard work, and turned it into a therapy that is now authorized at the European Union level. It is further confirmation that it is possible to create innovative research in Italy, and that the pharmaceutical sector in our country is globally competitive," explains Sergio Dompé, President of Dompé Pharmaceuticals. "At this time, my thoughts and gratitude go out to all of the Researchers, who have participated in so many countries and in various roles in the project, making an essential contribution to the creation of this new drug, but especially to Professor Rita Levi Montalcini; this research was born out of her brilliant intuition."

The process of registering the drug is underway, also in the United States, where cenegermin is still an investigational product, and where Dompé has started the submission to the Food and Drug Administration (FDA) of the first part of the registration application (Biologics License Application) through a procedure called "rolling submission." Finally, in Japan dialogue has begun with the Pharmaceuticals and Medical Devices Agency (PMDA) to define a development plan in the same direction.

About Dompé  

Dompé is one of the leading biopharmaceutical companies in Italy. It focuses on the development of innovative therapeutic solutions for diseases with a high social impact for which therapeutic options are lacking. Based in Italy, Dompé has its headquarters in Milan. Its research efforts focus on unmet therapeutic needs such as diabetes, organ transplantation, ophthalmology and oncology. The industrial pole of L'Aquila (Abruzzo) is home to a world class biotechnology plant developing drugs for Primary Care for the markets of about 40 countries worldwide. Dompé has its offices also in Albania, France, Germany, Great Britain, Spain and United States (Boston).

Forward looking statements 

This press release makes reference to certain information that may not coincide with expected future results. Dompé firmly believes in the soundness and reasonableness of the concepts expressed. However, some of the information is subject to a certain degree of indetermination in relation to its research and development activities and the necessary verifications to be performed by regulatory bodies. Therefore, as of today, Dompé cannot guarantee that the expected results will be consistent with the information provided above. 

1. M. Sacchetti, and A. Lambiase, Diagnosis and management of neurotrophic keratitis. Clin Ophthalmol 8 (2014) 571-9.
2. R. Levi Montalcini, The nerve growth factor 35 years later, Science 1987