FDA grants orphan drug designation to biologic therapy for Angelman syndrome

The FDA has granted orphan drug designation to a microbiome-modifying biologic to treat Angelman syndrome, according to the manufacturer.

In a press release, Biom Pharmaceutical Corp. stated its formulation of Bi104, a novel therapeutic, aims to modulate the microflora and metabolite profile in the gut to reduce neuroinflammation in patients with the disease.

Angelman syndrome is a rare neurodevelopmental disorder characterized by developmental delay, intellectual disability, speech impairment, and motor abnormalities which affects approximately 1 in 20,000 individuals worldwide. Currently, there is no specific treatment approved for preventing or controlling the condition.

“There is no doubt that we need innovative new approaches for these difficult-to-treat epilepsies,” Bobban Subhadra, MS, PhD, co-founder and president of Biom Pharmaceutical, said in the release. “Traditionally, these indications were overlooked and were underfunded, but a new wave of innovative drug development is in progress with gene therapy and siRNA-based technologies. I think modulating the gut microbiome is another novel treatment approach.”