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AGTC receives orphan medicinal product designation for retinitis pigmentosa gene therapy in Europe
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The European Commission has granted orphan medicinal product designation to an investigational gene therapy for the treatment of X-linked retinitis pigmentosa caused by mutations in the RPGR gene, according to a press release from Applied Genetic Technologies Corporation.
“Receiving orphan medicinal product designation from the [European Commission] for our XLRP gene therapy candidate is an important milestone for AGTC and a positive step forward in our ongoing efforts to develop effective treatments for rare inherited retinal diseases,” Sue Washer, president and CEO of AGTC, said in the release.
The company was previously granted orphan drug designation from the European Commission and the FDA for its gene therapy product candidates for the treatment of X-linked retinoschisis and the treatment of achromatopsia caused by mutations in the CNGA3 and CNGB3 genes.