Nightstar Receives U.S. and European Orphan Drug Designation for Gene Therapy to Treat Choroideremia

PRESS RELEASE:

London, 11 January 2015 – 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.

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The Choroideremia Research Foundation Inc. is an international, non-profit organization dedicated to raising funds to find a treatment or cure for Choroideremia, a rare inherited retinal degenerative disease that causes blindness.

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