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Posts Tagged ‘Retinitis Pignenotsa’

Dompe’ Receives FDA orphan drug designation for rhNGF in the treatment of Neurotrophic Keratitis (NK).

Reporter: Stephen J Williams, PhD

 

The U.S. FDA granted Dompe’ an orphan drug designation for rhNGF (recombinant human nerve growth factor) in the treatment of Neurotrophic Keratitis (NK).

Neurotrophic Keratitis (NK) is a rare, degenerative corneal disease caused by an impairment of corneal innervation (the distribution or supply of nerves), leading to a decrease or absence of corneal sensation and dysfunction of the corneal epithelium and abnormal corneal epithelial healing. The development of persistent epithelial defects or corneal ulcers can result in vision loss.

Severe NK is consistently recognized by clinicians as a serious condition lacking a highly effective treatment option.

The epidemiology of NK has not been well-defined. The estimated prevalence of patients with moderate-to-severe NK (stage 2-3) is less than 1 person in 5,000 globally.

Clinical trials in the U.S. are expected to begin in the next few months in leading research centers.

Dompé will be present at the American Association of Ophthalmology Annual meeting (Chicago, October 18-21). Currently, the enrollment is ongoing for the company’s Phase II trial with rhNGF in the treatment of NK.
Background – Dompé and its R&D

  • Dompé is a leading Italian biopharmaceutical company (with headquarters in Milan) committed to the development of innovative treatment solutions for rare, often orphan, diseases that have a high social impact, in areas where unmet treatment needs still exist.
  • The Company focuses its R&D activities in diabetes, ophthalmology, oncology and organ transplants.
  • The R&D activities are carried out in the Dompé biotech plant located in L’Aquila (Abruzzo), which has an internationally recognized expertise in the field of rare diseases.  
  • This year (2014), Dompé opened an office in New York, staffed with scientists and R&D teams in order to carry out and coordinate the scientific activities in the U.S.

 

Dompé commitment in ophthalmology – rhNGF

  • In ophthalmology, Dompé is promoting the research and development of Nerve Growth Factor (NGF), a soluble protein that stimulates the growth, maintenance and survival of neurons, whose discovery led to Prof. Rita Levi Montalcini being awarded the Nobel Prize in 1986.
  • Recombinant human Nerve Growth Factor (rhNGF) has been studied and produced exclusively at Dompé’s production site in L’Aquila, Italy, and is undergoing an international Phase II trial, called “REPARO”, to evaluate its efficacy and safety in the treatment of Neurotrophic Keratitis, a rare orphan disease. The trial is being conducted in 39 centers and nine European countries.

The medicine recently has been designated an orphan drug for the treatment of Retinitis Pigmentosa (RP), a severe, genetic rare disease that can lead to blindness for which there is currently no treatment available. A clinical trial in the EU, involving patients with RP, started in the first quarter of 2014 with the enrolment of the first patient.

SOURCE

From: Gail Thornton <gailsthornton@yahoo.com>
Reply-To: Gail Thornton <gailsthornton@yahoo.com>
Date: Wed, 23 Jul 2014 07:02:05 -0700
To: Aviva Lev-Ari <avivalev-ari@alum.berkeley.edu>
Subject: Dompe’ Receives FDA orphan drug designation for rhNGF

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