London, 4 May, 2020 – 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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Freeline is a privately-held clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Our vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-time curative treatment. Freeline is headquartered in the UK and has operations in Germany and the US.
FLT190, is an investigational liver-directed adeno-associated viral (AAV) gene therapy for the treatment of Fabry Disease. We believe the programme is the first clinical-stage AAV gene therapy international study in Fabry Disease. FLT190 is an in-vivo gene therapy administered by a one-time intravenous infusion.
The study, named MARVEL-1, is a multi-centre, international, dose-finding Phase 1/2 study in adult males with classic Fabry Disease. The study is focused on assessing the safety of FLT190 and its ability to transduce liver cells to produce continuous high levels of αGLA. In addition to safety, endpoints in the study include clearance of Gb3 and LysoGb3 from the plasma and urine, baseline renal and skin biopsies (repeated in long term follow up), renal and cardiac function, αGLA immune response, viral shedding and quality of life.
About Fabry Disease
Fabry Disease is an inherited, X-linked disease characterised by the progressive accumulation of glycosphingolipids in lysosomes throughout the body. It is caused by mutations in the gene encoding of the α-galactosidase A enzyme (αGLA) responsible for the breakdown of globotriaosylceramide (Gb3), a type of glycosphingolipid.
The condition ranges from mild to severe and may appear anytime from childhood to adulthood. The progressive accumulation of Gb3 leads to organ damage, major disability and often early mortality. Symptoms and signs include neuropathic pain, impaired sweating, gastrointestinal symptoms, renal failure, heart disease and increased risk of stroke. Current treatment consists of Enzyme Replacement Therapy and chaperone therapy to temporarily clear Gb3 accumulation and alleviate symptoms.
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