European Medicines Agency Approves Orphan Product Designation as Potential Gene Therapy for FA

The European Medicines Agency (EMA) has granted orphan medicinal product designation to Agilis Biotherapeutics’ gene therapy candidate AGIL-FA, being developed for the treatment of Friedreich’s ataxia (FA).

FA is a neurodegenerative disease caused by a defect in the frataxin (FXN) gene that reduces production of the frataxin protein. AGIL-FA is a gene therapy developed by Agilis in partnership with Intrexon to restore lost function in the FXN gene. AGIL-FA is delivered into the central nervous system (CNS) using adeno-associated virus technology.

Agilis completed several studies with AGIL-FA, including assessments in inducible pluripotent stem cell systems, a genetic model of FA, and several large studies of animals.

The clinical program for AGIL-FA is being developed in close collaboration with the Friedreich’s Ataxia Research Alliance (FARA), to guarantee its focus on real patient needs.

“FARA is delighted to continue our support of Agilis and their innovative approach to the treatment of FA,” Jennifer Farmer, executive director of FARA, said in a press release. “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 approval follows a Committee for Orphan Medicinal Products (COMP) positive opinion issued in July 2017 and an orphan drug designation granted by the U.S. Food and Drug Administration (FDA) last year. The company is currently in pre-investigational new drug (pre-IND) meetings with the FDA and is planning to apply in 2018.

“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.”

An orphan designation is granted to drugs meeting unaddressed medical needs in rare diseases, or diseases that affect less than five in 10,000 people in the European Union.

The regulatory title provides development and commercial incentives, such as 10 years of market exclusivity in the European Union, prioritized consultation with EMA specialists, expedited clinical trial review and certain reductions (or full exemptions) in regulatory fees.