Dompé Announces the Food and Drugs Administration (FDA) Has Granted Orphan Drug Designation to its rhNGF-based …

MILAN--(BUSINESS WIRE)--

Domp, the 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) discovered by Nobel Laureate Professor Rita Levi Montalcini has been granted orphan drug designation by the US FDA for the treatment of retinitis pigmentosa (RP). This gives new hope for the treatment of this disease for which at present there is no cure.

The orphan drug designation by the FDA comes just a few months after orphan drug designation was granted by the European Medicine Agency (EMA) and marks another milestone for this investigational drug, which is already in late-stage clinical development for the treatment of neurotrophic keratitis, a severe corneal disease.

We are particularly proud of this achievement: the orphan drug designation granted by the FDA is an important recognition for our company which is increasingly committed to producing innovation globally as testified by our direct presence in the US where we recently opened a subsidiary said Eugenio Aringhieri, CEO of the Domp Group We are strictly determined in discovering and developing prime high-tech drugs that can cure rare diseases, like retinitis pigmentosa, for which at present there are no treatment options. Our efforts are driven by our commitment to respond to the unmet needs of patients around the world based on our ability to deliver safe and effective solutions in sensitive medical areas.

Domp follows the development of rhNGF all the way, from production to clinical development. Building on initial results from the administration of the murine protein to a limited number of people with neurothropic keratitis, the company launched a dedicated project at its R&D sites to ensure the production of a drug based on recombinant human NGF (rhNGF). From that first indication, the company moved to synthesize the recombinant human protein and then assess the potential of the drug, which is entirely produced at Domp sites, also for the treatment of retinitis pigmentosa.

In the coming month a clinical trial will start to assess the efficacy and safety of rhNFG as treatment for retinitis pigmentosa. 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, Domp. 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 RhNGF RhNGF, the recombinant human nerve growth factor developed by Domp, 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. The recombinant human NGF may improve survival of retinal cells, slow down disease progression and help preserve patients vision.

About Retinitis Pigmentosa 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 globally, well below the threshold set for orphan drugs. As the treatment developed by Domp is being granted orphan drug designation by the FDA, in the US 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.

About Domp Domp is a leading biopharmaceutical company in Italy focused on developing innovative treatment solutions for rare, often orphan, diseases. Domp 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. Domp is based in Italy with HQ in Milan. Its industrial site in LAquila (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, Domp 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: http://www.dompe.com

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Dompé Announces the Food and Drugs Administration (FDA) Has Granted Orphan Drug Designation to its rhNGF-based ...