Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the CNS, announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to Agilis’ gene therapy product candidate, AGIL-FA, being developed for the treatment of Friedreich’s ataxia (FA), an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death.

Agilis is the first company to receive orphan designation from the FDA for a gene therapy to treat FA and it is the fourth time this year that Agilis has been granted orphan designation in the US and Europe for its gene therapies. The company’s gene therapies for AADC deficiency and Angelman syndrome have previously received orphan status.

“Having the first gene therapy product candidate to receive orphan drug designation from the FDA for the treatment of FA is an honour,” said Mark Pykett, president and CEO of Agilis. “The orphan designation is another step on our path to bringing 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 delivered with adeno-associated virus technology.

“We are extremely pleased to receive this landmark designation, as we move our FA development program forward,” said Christopher Silber, MD, Agilis chief medical officer. “With each of our IND and clinical stage pipeline candidates having now received Orphan Designation, this achievement highlights our efforts to advance innovative therapeutics for patients with rare genetic diseases affecting the CNS.”

Friedreich’s 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. Agilis’ FA gene therapy program 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.”

FDA Orphan Drug Designation is evaluated for drugs from all classes (e.g. small molecules, proteins, and gene or cell therapies) that are intended for the treatment of rare diseases, defined as diseases affecting fewer than 200,000 people in the United States. The designation provides sponsors with development and commercial incentives, including seven years of market exclusivity in the US, prioritized consultation by FDA on clinical studies, and certain exemptions from or reductions in regulatory fees.