Corporate Banner
Satellite Banner
Medicinal Chemistry
Scientific Community
Become a Member | Sign in
Home>News>This Article

ReNeuron Receives European and US Orphan Drug Designation

Published: Friday, August 30, 2013
Last Updated: Friday, August 30, 2013
Bookmark and Share
Company has received Orphan Drug Designation for its ReN003 stem cell therapy candidate for retinitis pigmentosa.

ReNeuron Group plc has announced that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone.

The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.

Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating.

Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.

ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying.

Retinitis pigmentosa affects approximately 4 in 10,000 people in the EU and US.

The late pre-clinical development of the ReN003 programme also benefits from a recently awarded £0.8 million Biomedical Catalyst grant from the UK Technology Strategy Board.

The Company is targeting an initial Phase I/II clinical trial with ReN003 in the UK and the US, in retinitis pigmentosa patients, with clinical trial applications expected to be filed in the middle part of 2014.

Michael Hunt, Chief Executive Officer of ReNeuron, said: “We are delighted to have received Orphan Drug Designation for our ReN003 stem cell therapy candidate for retinitis pigmentosa from both the European Commission in Europe and the FDA in the US. Orphan status diseases are an increasing area of therapeutic and commercial focus by the mainstream pharmaceutical industry due to the distinct regulatory and market exclusivity advantages that Orphan Drug Designation confers. This fact bodes well for the commercial potential of our ReN003 therapy.”

Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,800+ scientific posters on ePosters
  • More than 4,000+ scientific videos on LabTube
  • 35 community eNewsletters

Sign In

Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Scientific News
Nanocarriers May Carry New Hope for Brain Cancer Therapy
Berkeley lab researchers develop nanoparticles that can carry therapeutics across the brain blood barrier.
Potential Persistent Tuberculosis Treatment
Researchers have discovered several first-in-class compounds that target hidden TB infections by attacking a critical process the bacteria use to survive in the hostile environment of the lungs.
Shedding Light on “Dark” Cellular Receptors
UNC and UCSF labs create a new research tool to find homes for two orphan cell-surface receptors, a crucial step toward finding better therapeutics and causes of drug side effects.
Giant Molecules Inhibit Ebola Infection
European researchers have designed a "giant" molecule formed by thirteen fullerenes covered by carbohydrates which, by blocking this receptor, are able to inhibit the cell infection by an artificial ebola virus model.
First Therapy Appearing to Reverse Decline in Parkinson’s
An FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson’s disease and Lewy body dementia in a small clinical trial, say researchers at Georgetown University Medical Center (GUMC).
Fighting Pain with Ketamine
Researchers at the Texas A&M Health Science Center are using ketamine, a drug that already exists as an anesthetic, to treat pain.
Possible New Treatment for Neurodegenerative Diseases Found
Researchers at the University of Liverpool have found that a well-established anti-epileptic drug could also be used as a treatment for neurodegenerative diseases.
Breast Cancer Drug Beats Superbug
Tamoxifen helps white blood cells clear multidrug-resistant bacteria in lab and mouse studies.
Have Your Drug Nano-Delivered Via Microbubble
A UC engineering professor is working to develop effective nanoparticle-bubble drug delivery systems to access precise locations in the body to treat medical conditions such as cancer, eye disease and spinal disc degeneration.
Atriva Therapeutics GmbH Develops Innovative Flu Drug
Highly effective against seasonal and pandemic influenza.
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,800+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,000+ scientific videos