Pharma Focus Europe

OrphAI Therapeutics Obtains Orphan Drug Designation in the European Union for AIT-101 as a Treatment for Amyotrophic Lateral Sclerosis

Friday, February 16, 2024

OrphAI Therapeutics Inc., a biopharmaceutical firm focused on developing novel therapies for rare diseases, has announced that it has obtained Orphan Drug Designation (ODD) from the European Union (EU) for its drug AIT-101, intended for the treatment of amyotrophic lateral sclerosis (ALS). This designation follows a similar ODD granted by the U.S. FDA in June 2023.

According to Dr. Brigette Roberts, CEO and Director of OrphAI, this dual orphan designation represents a significant milestone in bringing AIT-101 to ALS patients. Dr. Roberts highlighted the drug's potential in clearing toxic aggregates associated with ALS and other neurodegenerative diseases, expressing anticipation for advancing AIT-101 into further clinical development stages.

AIT-101 functions as a potent and highly selective inhibitor of the lipid kinase PIKfyve. Its inhibition leads to the activation of the transcription factor TFEB, thereby enhancing the clearance of toxic protein aggregates through the lysosomal autophagy pathway. Preclinical studies have shown promising results, including the reduction of aggregates in human subjects, improved survival of motor neurons in ALS models, and the mitigation of functional deficits in ALS animal models with mutant TDP-43. AIT-101 is currently the most advanced PIKfyve inhibitor in clinical development.



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