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FDA, EMA grant Nightstar orphan drug designation for gene therapy to treat choroideremia
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Nightstar has received orphan drug designation from the FDA and the European Medicines Agency for a gene therapy to treat choroideremia, according to a press release.
The designation will provide regulatory support in development activities such as protocol assistance, reduced fees, tax incentives and market exclusivity for the product upon approval, the release said.
“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,” David Fellows, CEO of Nightstar, said in the release.
In a study published in The Lancet, six patients showed subjective improvement in dim light and two of the six patients were able to read more than two lines on an eye chart 6 months after the gene therapy treatment, the release said.