Committee for Medicinal Products for Human Use (CHMP) issues positive recommendation for approval of first treatment (givosiran) for acute hepatic porphyria (AHP)

Approval based on RCT (n=94) of AHP patients who had ≥ 2 attacks in past 6 months. Givosiran, a short synthetic strand of RNA that interferes with production of haem, resulted in significant decrease of annual attacks, less pain and improved quality of life.


European Medicines Agency