‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access
As it looks to emerge from bankruptcy, Mallinckrodt (MNK) has sold an experimental medicine that until recently was being tested to treat a fatal genetic disorder. But the company has said it will work with the buyer to ensure it remains available to children, an unexpected twist in the latest controversy over access the drug.
At issue is a medication called adrabetadex, which the company was studying to combat Niemann-Pick type C, or NPC, a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances inside of cells. The life expectancy of someone with the condition is about 20 years, but can be much shorter if NPC emerges during infancy.
