Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation for FLT190 for the treatment of Fabry Disease. This comes soon after Freeline announced it had received Orphan Drug Designation for FLT190 from the European Commission, based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency.
“Receiving Orphan Drug Designation from the FDA is another step forward for the development of FLT190 for patients with Fabry Disease," said Chris Hollowood, Chairman of Freeline." Fabry Disease has a wide spectrum of symptoms that can have a devastating impact on people’s lives and we believe that FLT190 has the potential to be a functional cure that can halt progression of the disease and address many of these serious symptoms."
Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 individuals in the United States. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax credits, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval.
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