LGMD Gene Therapy ATA-100 Wins Orphan Drug Status From FDA, EMA

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by Margarida Maia PhD |

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ATA-100 named orphan drug | Muscular Dystrophy News | announcement illustration of woman with megaphone

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ATA-100, a one-time gene therapy being developed by Atamyo Therapeutics for a specific form of limb-girdle muscular dystrophy (LGMD) called 2I or R9 (LGMD2I/R9).

In Europe, the therapy also was named an orphan drug by the European Medicines Agency (EMA) for the treatment of LGMD.

With these designations, Atamyo Therapeutics can benefit from seven and 10 years of exclusive rights to market the therapy in the U.S. and Europe, respectively. Other benefits include tax credits, access to research grants, and scientific advice on the most appropriate way to generate evidence on the therapy’s benefits and risks.

Additionally, the company was given the green light by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) late last year to start testing ATA-100 in humans for the first time. Now, it has been given an added go-ahead by the Danish Medicines Agency (DKMA) to start a clinical trial.

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“We are thrilled by this additional approval for our first-in-human trial with ATA-100 and by the Orphan Drug Designation in the United States and in Europe” Stéphane Degove, CEO of Atamyo, said in a press release.

“This single-administration treatment brings hope to patients with LGMD2I/R9,” Degove said.

Atamyo also was awarded public financing of €2 million (about $2.2 million) by Bpifrance to get its first clinical trial of ATA-100 off the ground. This funding is part of the Deeptech Development Aid program, a French government initiative to help start-ups get financed in France, where an additional clinical trial application for ATA-100 is currently under review.

“The strong financial support by the French government will reinforce the rapid progress towards a first administration of ATA-100 in patients,” Degove said.

LGMD2I/R9 is caused by mutations in FKRP, a gene that provides instructions to make a protein called fukutin-related protein (FKRP). The first symptoms appear in childhood or early adulthood and progress slowly, causing muscle weakness that can affect the ability to walk and breathe.

ATA-100 is expected to work by delivering a healthy copy of the FKRP gene to cells. The gene is carried in a harmless vector that the company has modified based on a naturally occurring virus called adeno-associated virus (AAV).

The therapy is based on earlier work by Isabelle Richard, PhD, chief scientific officer of Atamyo and head of the Progressive Muscular Dystrophies Laboratory at Genethon, the company from which Atamyo was spun off.