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Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich AtaxiaAgilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that the European Commission (EC) has granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to the Company's gene therapy product candidate, AGIL-FA, being developed for the treatment of Friedreich ataxia (FA), an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death. The EC's approval follows a positive opinion in July 2017 from the European Medicine Agency's (EMA (News - Alert)) Committee for Orphan Medicinal Products (COMP). This follows the Orphan Drug Designation for AGIL-FA granted by the U.S. Food and Drug Administration (FDA) last year. The Company's gene therapies for AADC deficiency and Angelman syndrome have previously received orphan status in both the EU and US. "Receiving the first orphan designations for a gene therapy product candidate from the FDA and now the EU for the treatment of FA is an honor," said Mark Pykett, President and CEO of Agilis. "The orphan designation is another step on our path to bring this important new therapy to patients who currently lack treatment options." AGIL-FA is a gene-therapy product consisting of a unique gene construct developed in partnership with Intrexon Corporation (NYSE: XON) delivered with adeno-associated virus technology. The Company has completed extensive characterization of AGIL-FA, including analyses in inducible pluripotent stem cell systems, a genetic model of FA, and multiple large animal studies demonstrating reproducible transduction of the FXN gene and expression of frataxin protein in target CNS cells integral to the neurological manifestations in FA in support of human clinical studies. The Company has completed a Pre-IND meeting with the FDA and is on track to open an IND in 2018. "We are extremely pleased to receive this designation, as we move our FA development program forward," said Kirsten Gruis, MD, Agilis' Chief Medical Officer. "With each of our IND and clinical stage pipeline candidates having now received Orphan Deignation in two major markets, our programs are well positioned to advance innovative therapeutics for patients with rare genetic diseases affecting the CNS." Friedreich ataxia (FA) is a rare and life-shortening neurodegenerative disease caused by a defect in the FXN gene that reduces production of the frataxin protein. AGIL-FA is focused on delivering corrective DNA to specific CNS cells to restore frataxin protein levels. Agilis has worked closely with the Friedreich's Ataxia Research Alliance (FARA) to focus the development program on patient needs. "FARA is delighted to continue our support of Agilis and their innovative approach to the treatment of FA," said Jennifer Farmer, MS, Executive Director of FARA. "We look forward to continuing our partnership to advance this important potential therapy, as well as supporting research to identify biomarkers and clinical outcome measures, which will advance the development of the product candidate into clinical trials." The EU orphan designation provides Agilis with development and commercial incentives, including 10 years of market exclusivity, prioritized consultation by EMA on the development of the drug, including clinical studies, and certain exemptions from or reductions in regulatory fees in Europe. Orphan designation is granted to drugs that are intended for the treatment of life threatening or chronically debilitating rare diseases where no therapeutic options either exist or are satisfactory. Rare diseases are those defined as having a prevalence of less than five in 10,000 people in the European Union.
About Friedreich ataxia Visit www.curefa.org for more information.
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