Navigation Links
Dompe announces the Food and Drug Administration (FDA) has granted orphan drug designation to its rhNGF-based treatment for neurotrophic keratitis

NEW YORK, July 23, 2014 /PRNewswire/ -- Dompe, a leader in R&D for new therapeutic solutions for the treatment of rare diseases, announces that rhNGF (Recombinant Human Nerve Growth Factor) has been designated as an orphan drug by the American Food and Drug Administration (FDA).

The candidate drug, developed by the research of Dompe, has been designated as an orphan drug for the treatment of neurotrophic keratitis, a degenerative corneal disease that affects less than 1 in 5,000 people1 and is currently without a cure. This is the second orphan drug designation by the FDA for rhNGF, after the recent designation for the treatment of retinitis pigmentosa, a rare genetic disease that affects more than 1 million people worldwide2.

This acknowledgment represents an important step in the clinical development of the drug, which is currently in advanced stages of experimentation for the treatment of neurotrophic keratitis in the REPARO study. The randomized, double-blind study includes 39 centers in 9 European countries (Italy, Great Britain, Germany, France, Belgium, Spain, Portugal, Poland, Hungary) and involves patients suffering from unilateral neurotrophic keratitis with grade 2 lesions (persistent epithelial defect) or grade 3 lesions (corneal ulcers) that do not respond to the currently available medical treatment. The objective of the trial is to evaluate the safety, tolerability, and effectiveness of two different doses of recombinant human nerve growth factor (rhNGF) compared to the placebo. The more than 170 patients enrolled in the study are divided into three different groups, treated respectively with two different doses of rhNGF and with the placebo. The secondary objectives include the evaluation of corneal lesion healing, improvement in visual acuity, and the 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," - explains Eugenio Aringhieri, CEO of the Dompe Group. "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."

rhNGF is the result of a specific research project at the Dompe Center for Research & Development of l'Aquila and is produced using recombinant DNA technology, or by transferring human genetic material into bacterial DNA, enabling the bacteria to produce NGF that is entirely identical to that naturally produced by the human body.

About rhNGF

rhNGF, the Recombinant Human Nerve Growth Factor developed by Dompe, is identical to the protein that is naturally produced by the body and promotes the development and survival of nerve cells, including those of the retina. NGF (Nerve Growth Factor) was discovered by Rita Levi Montalcini, who was awarded the Nobel Prize in Physiology or Medicine in 1986.  The protein stimulates the growth, maintenance, and survival of nerve cells, in addition to the cells of the cornea and the retina. The cornea in particular has a particularly dense innervation, approximately 400 times denser than that of the skin. Treatment with rhNGF-based ophthalmic solution aims to restore the innervation of the corneal area that has been compromised by the disease in order to promote recovery of visual function. In the future, rhNGF may also prove useful in other forms of optic neuropathies, as it has for retinitis pigmentosa in the treatment of glaucoma and dry eye syndrome due to its innovative mechanism of action.

Neurotrophic keratitis

Neurotrophic keratitis is a rare serious degenerative disease of the eye, for which there is currently no cure, characterized by progressive damage to the cornea that can lead to corneal perforation, with consequent loss of vision. It can be caused by various clinical ocular conditions such as herpetic corneal infections (herpes zoster and simplex), eye surgeries, misuse of topical medications, use of contact lenses, and even systemic conditions such as diabetes or vitamin A deficiency. It affects less than 1 in 5,000 people in the world.

About Dompe

Dompe is one of the top biopharmaceutical companies in Italy, focused on the development of innovative pharmaceutical solutions for diseases with high social impact, which are often incurable.  Based out of Italy, Dompe has its headquarters in Milan and focuses its research efforts in areas with unmet therapeutic needs, such as diabetes, organ transplants, ophthalmology, and oncology. The industrial center in L'Aquila (Abruzzo) houses a biotechnology facility known worldwide for its excellence and develops Primary Care drugs intended for markets in 30 countries worldwide. In 2013, Dompe opened a subsidiary in the United States.

For more information:

Forward looking statements

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

1 Sacchetti M., Lambiase A., Diagnosis and management of neurotrophic keratitis. Clinical Ophthalmology 2014; 8: 571–579.

2 Source: Anasagasti A, Irigoyen C, Barandika O et al., Current mutation discovery approaches in retinitis pigmentosa, Vision Res. 2012; 75: 117-129.

Logo -

Copyright©2014 PR Newswire.
All rights reserved

Related medicine technology :

1. Pelvalon Announces Results from the LIFE Pivotal Trial at the 2014 AUGS/IUGA Conference
2. ArmaGen Announces Strategic Licensing and Collaboration Agreement with Shire to Develop AGT-182 for Treatment of Hunter Syndrome
3. Galmed Announces FDA Clearance of IND of Armachol for the Treatment of Fatty Liver Disorders
4. Quantum Materials Announces New Outside Directors and Adds Scientific Advisory Board
5. Avanir Pharmaceuticals Announces FDA Acceptance of IND for AVP-786 for the Adjunctive Treatment of Major Depressive Disorder
6. WaferGen Bio-Systems Announces Appointment of Ivan Trifunovich as Chairman of the Board; Joel Kanter to Serve as Lead Independent Director
7. Radyalis Announces Next Generation Of Monte Carlo Proton Therapy Planning For Advanced Radiation Cancer Treatment
8. Richmond Private Equity Group announces joint venture with Taking You Forward Inc.
9. Depomed Announces District Court Order in ANDA Litigation
10. Amgen Announces Positive Phase 3 Results for AMG 416 for the Treatment of Secondary Hyperparathyroidism in Patients with Chronic Kidney Disease Receiving Hemodialysis
11. JDRF Partner ViaCyte Announces Key Project Milestone to Advance Innovative Encapsulated Cell Replacement Therapy Product for Type 1 Diabetes
Post Your Comments:
(Date:11/27/2015)... , November 27, 2015 ... set to go online. The potential to save costs, ... vast and far from fully exploited as yet. Here, ... health records, either via mobile tablet or directly at ... --> ) -->      ...
(Date:11/27/2015)... 2015 ... "2016 Global Tumor Marker Testing Market: ... Sales Segment Forecasts, Innovative Technologies, Instrumentation Review, ... their offering. --> ) ... "2016 Global Tumor Marker Testing Market: Supplier ...
(Date:11/26/2015)... the Netherlands , November 26, 2015 ... A new combination approach blends immunotherapy with ... A new combination approach blends immunotherapy with Bremachlorin-photodynamic ... A new combination approach blends immunotherapy with Bremachlorin-photodynamic ... the Netherlands has found that immunotherapy can ...
Breaking Medicine Technology:
(Date:11/27/2015)... (PRWEB) , ... November 27, 2015 , ... ... the November 27th edition of USA Today in Atlanta, Dallas, New York, Minneapolis, ... of 750,000. The digital component is distributed nationally, through a vast social media ...
(Date:11/27/2015)... ... 2015 , ... Consistent with the Radiology Business Management ... Radiology Marketing Programs meeting will showcase some of the best 2015 radiology ... Palace in Las Vegas with a pre-conference session on a collaborative approach for ...
(Date:11/27/2015)... Minn. (PRWEB) , ... November 27, 2015 , ... ... start failing. Secura Consultants has prided itself for not only fulfilling the needs ... income protection solutions at an affordable price and providing top-tier customer service. However, ...
(Date:11/27/2015)... ... November 27, 2015 , ... The men and women ... healthcare organizations in the country. They have overseen financial turnarounds, shown commitment to ... the healthcare industry as a whole through their advocacy and professional efforts. , ...
(Date:11/27/2015)... ... November 27, 2015 , ... Indosoft Inc., developer ... incorporation of Asterisk 11 LTS (Long Term Support) into its Q-Suite 5.10 product ... Q-Suite 5.10 up-to-date with a version of Asterisk that will receive not only ...
Breaking Medicine News(10 mins):