US FDA approves Mirum's drug for rare bone disorder
MIRM•The FDA approved Mirum Pharmaceuticals' once-daily oral drug Atebrioz for patients aged 12 and older with fibrodysplasia ossificans progressiva; Mirum expects to launch it in October and plans to announce its price then.
1. FDA approves treatment
The FDA approved Mirum Pharmaceuticals' zilurgisertib, branded as Atebrioz, for patients aged 12 and older with fibrodysplasia ossificans progressiva (FOP), a genetic disorder that causes bone to form in muscles, tendons and ligaments, restricting movement and leading to severe disability.
2. Drug and launch details
Atebrioz is a once-daily oral treatment that blocks ALK2, a protein abnormally active in most patients with FOP that drives bone formation outside the skeleton. The recommended dose is 100 milligrams. Mirum expects to launch the drug in October and plans to announce its price at launch, CEO Chris Peetz said.
