- The European Medicines Agency (EMA) designates Arrowhead Research's (ARWR -1.1%) RNAi therapeutic candidate, ARC-AAT, an Orphan Drug for the treatment of congenital alpha-1 antitrypsin (AAT) deficiency, a rare genetic disorder that leads to lung and liver problems. AAT is a protein that protects the lungs.
- Among the benefits of Orphan Drug status in the EU is a 10-year period of market exclusivity for the indication, if approved.
- The FDA designated ARC-AAT an Orphan Dug for AAT deficiency last June.
- Previously: Arrowhead Research's ARC-AAT an Orphan Drug for rare genetic liver disease (June 10, 2015)
Arrowhead's ARC-AAT an Orphan Drug in Europe for rare genetic disorder that causes lung and liver disease
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