NightstaRx Ltd ("Nightstar"), the biopharmaceutical company specialising in bringing therapies for retinal dystrophies to patients, has received both U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) Orphan Drug Designation for its lead programme, a gene therapy to treat Choroideremia, an X-linked recessive disorder that leads to progressive blindness.
Orphan Drug Designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including regulatory support in development activities such as protocol assistance, reduced fees, tax incentives and several years of market exclusivity for the product upon regulatory approval.
David Fellows, CEO of Nightstar said:
"We are delighted to have received Orphan Drug Designation for our gene therapy treatment for Choroideremia. We are committed to finding effective treatments for retinal dystrophies and the support and assistance of the regulatory bodies in the U.S and Europe will be invaluable in our further development work."
The retinal gene therapy, initially developed by Professor Robert MacLaren at Oxford's Nuffield Laboratory of Ophthalmology, has shown very promising results which were published in The Lancet in January of 2014. The ground-breaking therapy involves injecting a small modified virus, AAV2.REP1 to deliver the correct version of the choroideremia (CHM) gene to cells in the retina of the eye. The Lancet reported that six months after treatment with this therapy, the first six patients showed subjective improvement in their vision in dim light and two of the six were able to read more lines on the eye chart.
Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or 'tunnel vision', and later a loss of central vision. Progression of the disease continues throughout the individual's life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.
For patients interested in participating in choroideremia trials please follow this link: http://www.eye.ox.ac.uk/research/clinical-ophthalmology-research-group/clinical-trials/choroideremia-gene-therapy