MILAN--(BUSINESS WIRE)--Dompé, a leading company in research & development of novel treatment solutions for rare diseases, announces its investigational drug based on the recombinant human nerve growth factor (rhNGF) for the treatment of retinitis pigmentosa (RP) has been granted orphan drug designation.
This marks another milestone in the development of the drug based on the discovery made by Nobel Laureate Professor Rita Levi Montalcini. Another drug discovered and developed by Dompé to improve the efficacy of pancreatic islet transplantation in diabetes patients, Reparixin, had already obtained Orphan Drug status from the European Medicine Agency (EMA) and the US Food and Drugs Administration (FDA).
The term retinitis pigmentosa denotes a group of hereditary eye diseases with progressive loss of vision. In patients suffering from this condition the light receptors, i.e. the retinal cells known as the rods and cones that convert light images to nerve signals and sends them to the brain, get damaged and progressively degenerate.
Currently retinitis pigmentosa affects about 3 in 10,000 people in the European Union, adding up to a total of some 153,000 patients. This number is well below the threshold set for orphan drugs, i.e. drugs to treat conditions that affect no more than 5 in 10,000 people. As the treatment developed by Dompé is being granted orphan drug designation, in the European Union there is no satisfactory targeted treatment for retinitis pigmentosa. People with this disorder receive just genetic counselling about the risk of transmitting the disease to their offspring and general support therapy.
RhNGF, the recombinant human nerve growth factor developed by Dompé, is the same type of protein as the one naturally produced by the human body that fosters development and survival of nerve cells including retinal cells. NGF has proven to be effective in inhibiting retinal degeneration in several animal models in retinitis pigmentosa. Supplementing the NGF produced by the human body, the recombinant human NGF may improve survival of retinal cells, slow down disease progression and help preserve vision.
“We are particularly proud of the orphan drug designation granted by the EMA, which spurs us to continue with our research and development of prime high-tech drugs that can cure rare diseases, like retinitis pigmentosa, for which at present there are no treatment options. – said Eugenio Aringhieri, CEO of the Dompé Group – ‘We will continue to leverage our ability to generate innovation to respond to the unmet needs of patients, as we are doing with Reparixin, that is currently in Phase III clinical development. Reparixin, too, was granted Orphan Drug Designation by the EMA and the US FDA, which is testament to the importance of this molecule and its potential of becoming a tangible treatment option for patients with type 1 diabetes who cannot keep it under control with the treatments that are currently available.”
Research on NGF started following observations of the effects this soluble protein of murine origin had on a group of people with neurotrophic keratitis (NK), which is a rare corneal disease. Based on initial clinical studies Dompé has developed a specific research project at its Research & Development Centre in L’Aquila (Italy) for the biotechnological production of a drug based on the recombinant human NGF. The Group’s production facilities in L’Aquila are the only one in the world producing this recombinant protein for eye treatments.
“The drug is produced using recombinant DNA techniques, specifically by transferring human genetic material into a bacterium which thus becomes capable of producing NGF – explains Marcello Allegretti, Chief Scientific Officer, Dompé. We are currently investigating rhNFG in the phase I/II clinical trial REPARO as treatment for neurotrophic keratitis, a rare eye disease which in its most severe forms affects nearly 1 in 10,000 people worldwide. Like Retinitis Pigmentosa, currently there is no specific treatment available for neurotrophic keratitis, which is a progressive degenerative disease of the cornea that may result in blindness.”
About Dompé
Dompé is a leading biopharmaceutical company in
Italy focused on developing innovative treatment solutions for rare,
often orphan, diseases.
Dompé pursues this goal also by proactively
promoting and leveraging networking of prime players along the entire
pharmaceutical supply chain – from R&D through manufacturing to
commercialisation.
Dompé is based in Italy with HQ in Milan. Its
industrial site in L’Aquila (Abruzzi, Italy) employs 230 people, of whom
70 in R&D, and features production facilities that meet the highest
industry standards in terms of both quality and technology, developing
drugs that are sold worldwide.
In 2012, the Dompé Group has
ploughed back over 10% of sales into R&D, focusing its commitment on
therapeutic areas with unmet medical needs, such as diabetes,
ophthalmology and oncology.
For more information: www.dompe.com
Sources:
- European Medicines Agency website: www.emea.europa.eu
Forward looking statements
This press release may contain forward looking statements. Dompé firmly believes the expectations reflected in forward looking statements are reasonable and built on sound assumptions. However, by their very nature, forward looking statements are subject to uncertainties including those inherent in R&D and those associated with decisions made by regulatory authorities. Hence, forward looking statements are not a guarantee of future results which may be different.
