The US Food and Drug Administration has approved Mirum Pharmaceuticals’ pill for a rare bone disorder, giving patients a new treatment option that can help slow the disease’s progression.
The once-daily drug zilurgisertib, sold under the brand name Atebrioz, has been approved for patients aged 12 years and older with fibrodysplasia ossificans progressiva, or FOP.
The condition causes muscles, tendons, and ligaments to gradually turn into bone, restricting movement and leading to severe disability.
Atebrioz works by blocking ALK2, a protein that is abnormally active in most patients with FOP and drives bone formation outside the skeleton. The recommended dose is 100mg.
FOP is usually diagnosed in early childhood. Mirum said about 300 people in the United States and 900 worldwide are living with the condition.
The company expects to launch the drug in October and said it will announce the price at that time.
Mirum CEO Chris Peetz said the medicine could be a game-changer for patients because it may help stop the progressive buildup of bone.
The FDA’s approval was based on a 63-patient study in which Atebrioz significantly reduced new bone formation compared with placebo at week 24.
Current treatment options for FOP include Ipsen’s oral drug Sohonos and Regeneron’s monthly infusion Pasatru.
Analysts said Atebrioz could become a preferred option because it combines oral dosing with strong efficacy and a favourable safety profile.
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