Orphan designation: Ornithine phenylacetate Treatment of acute liver failure, 20/02/2025 Positive
At the time of designation, acute liver failure affected approximately 0.06 in 10,000 people in the EU. This is equivalent to a total of around 3,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the …