Regulatory support for Choroideremia treatment from Nightstar

11th March 2015

Nightstar, a spin-out from the University of Oxford in 2014 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.

NightstaRx Ltd (“Nightstar”), which has raised £17 million including initial backing from Syncona, the venture arm of the Wellcome Trust, 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.

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.

For further see the Nightstar website.

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