Biompharma’s Bi104 Receives FDA Orphan Drug Designation for Angelman Syndrome Treatment

Bi104 is a microbiome-modifying biologic designed to diversify and modulate the gut microflora and metabolite profile.

BIOM Pharmaceutical Corporation has announced that its innovative drug, Bi104, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman Syndrome (AS). This designation is a significant milestone in the quest to find effective treatments for this rare neurodevelopmental disorder.

Angelman Syndrome, affecting approximately 1 in 20,000 individuals worldwide, is marked by developmental delays, intellectual disabilities, speech impairments, and motor abnormalities.

Currently, no approved treatments exist to prevent or control the syndrome; available options are limited to symptom management and supportive care.

Recent studies have highlighted the connection between microbiome imbalances and neurodegenerative diseases. Communication between gut microbiota and brain cells is bidirectional and influences the central nervous system’s physiology. Intestinal dysbiosis has been linked to various neurological disorders, such as autism spectrum disorders, multiple sclerosis, Parkinson’s disease, and Alzheimer’s disease.

Bi104, formulated by BIOM Pharma, is a microbiome-modifying biologic designed to diversify and modulate the gut microflora and metabolite profile. The goal is to reduce neuroinflammation in AS patients. Preclinical studies have demonstrated significant benefits in improving seizures and behavioral aspects in an established AS mouse model.


Dr. Bobban Subhadra, Co-Founder and President of BIOM Pharmaceutical Corporation, expressed excitement about this achievement: “There is no doubt that we need innovative new approaches for these difficult-to-treat epilepsies. Traditionally, these indications were overlooked and 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. In this regard, we are excited to get the orphan designation for Bi104, which will be a key catalyst for our effort to develop the clinical pathway for Bi104.”

Tom Gardner, Chairman of BIOM Pharmaceutical Corporation, added, “Procuring orphan designation for Bi104 is a major milestone for us and validates our technology and formulation to initiate a clinical program. This will also help us procure funding and strategic collaborations.”

BIOM Pharmaceutical Corporation is committed to advancing Bi104 through clinical development and regulatory approval processes, aiming to bring hope and relief to patients and families affected by Angelman Syndrome.