FDA grants orphan drug status to Aro’s therapy for Pompe disease

The US Food and Drug Administration (FDA) has granted Orphan drug designation for Aro Biotherapeutics’ ABX1100 for the treatment of Pompe disease.

Investigated Centyrin-small interfering ribonucleic acid (siRNA) conjugate, ABX1100 acts on the glycogen synthase 1 (Gys1) gene in muscle.

Gys1 is an enzyme that synthesizes glycogen in muscles. Inhibiting this enzyme has been found to reduce glycogen levels and is a new approach to treating Pompe disease.

In a mouse model of Pompe, ABX1100 was shown to significantly decrease Gys1 mRNA and GYS1 protein, causing a significant drop in skeletal muscle glycogen levels.

The company plans to enter ABX1100 into clinical trials in the middle of next year.

Aro Biotherapeutics Chief Medical Officer Mitty Doyle said, “We are delighted to have received this designation and are pleased with the FDA’s recognition of ABX1100’s potential to improve the lives of patients living with Pompe disease.

“We believe our new treatment approach has the potential to address the large unmet need that exists in Pompe disease, and we are excited to move ABX1100 into clinical trials next year.”

Biotech firm Aro Biotherapeutics focuses on developing tissue-targeted genetic treatments.

Pompe disease is a rare genetic disorder that causes debilitating muscle weakness that progresses over time.

It is caused by a mutation in the enzyme acid alpha-glucosidase (GAA), which breaks down glycogen in muscle.

Because of the mutation, patients with Pompe disease have elevated glycogen levels, leading to disease progression.

Currently, patients receive enzyme replacement therapy (ERT) with recombinant GAA administered intravenously.

Cell and gene therapy coverage for pharmaceutical technologies is supported by Cytiva.

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