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The investigational drug developed by Dompé gives new hope for the treatment of retinitis pigmentosa, for which at present there is no cure.

Dompé announces the Food and Drug Administration (FDA) has granted orphan drug designation to its rhNGF-based treatment for retinitis pigmentosa

  • Retinitis pigmentosa includes a group of hereditary eye diseases with progressive loss of vision for which currently there is no cure
  • Retinitis pigmentosa affects about 3 in 10,000 people in the US
  • Dompé, which recently opened a subsidiary in the US, is one of the leading biopharmaceutical company in Italy and a global player in the development of innovative treatment solutions for rare orphan diseases in ophthalmology

Milan, September 11th, 2013 – Dompé, 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 Dompé 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.”

Dompé 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 Dompé 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, 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.”