FDA grants orphan drug status for Arrowhead’s ARC-AAT to treat liver disease
AATD is a rare genetic disease that severely damages the liver and lungs of affected children and adults. Currently, the company is conducting part B of a Phase
The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have granted paediatric clinical trial waivers to Shanton Pharma for its lead candidate SAP-001, easing the drug’s regulatory paths to approval.
Based on achieving this critical milestone, Aspyrian Therapeutics recently submitted and has received FDA’s acceptance of an Investigational New Drug (IND) Application for RM-1929, their first-in-class, precision targeted