Rare Disease Report

European Commission Grants Orphan Drug Status to Friedreich Ataxia Treatment

OCTOBER 31, 2017
Mathew Shanley
This morning it was announced that the European Commission granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to Agilis Biotherapeutics’ gene therapy product candidate AGIL-FA, intended for the treatment of Freidreich ataxia (FA).

In August, the therapy was given a positive opinion from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), and in August, it was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).

Friedrich ataxia is the result of a mutation in the FXN gene and frequently starts to present in patients between the ages of 5 and 18 years. The gene is responsible for the production of the mitochondrial protein frataxin, and the autosomal recessive rare genetic condition can eventually lead to the gradual loss of muscle coordination, as well as heart disease and diabetes.

“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 in a press release. “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 one of Agilis’ three gene therapies to have received orphan drug status. It contains a unique gene construct that was developed in collaboration with Intrexon Corporation and delivered via adeno-associated virus technology.

“We are extremely pleased to receive this designation, as we move our FA development program forward,” said Kirsten Gruis, MD, Agilis’ Chief Medical Officer in a press release. “With each of our IND and clinical stage pipeline candidates having now received Orphan Designation in two major markets, our programs are well positioned to advance innovative therapeutics for patients with rare genetic diseases affecting the CNS.”

In 2018, the company is expected to file an Investigational New Drug (IND) application with the FDA. The two sides recently met for a Pre-IND meeting. Agilis is working closely with the Friedreich’s Ataxia Research Alliance (FARA) to focus the development program specifically on patients’ needs.

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