Key Takeaways
- The US FDA has approved Atebrioz for treating fibrodysplasia ossificans progressiva.
- Atebrioz works by blocking ALK2, a protein that drives bone formation outside the skeleton.
- The drug is expected to launch in October with a price to be announced.
The US Food and Drug Administration (FDA) has approved Atebrioz, a new drug developed by Mirum Pharmaceuticals, for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare bone disorder.
FOP causes muscles, tendons, and ligaments to gradually turn into bone, severely restricting movement and leading to disability.
Atebrioz, sold under the brand name Atebrioz, is a once-daily pill that works by blocking ALK2, a protein that is abnormally active in most FOP patients and drives bone formation outside the skeleton.
The drug has been approved for patients aged 12 years and older, with the recommended dose being 100mg per day.
The FDA’s approval was based on a 63-patient study where Atebrioz significantly reduced new bone formation compared to a placebo at week 24.
Currently, the only treatment options for FOP include Sohonos, an oral drug from Ipsen, and Pasatru, a monthly infusion from Regeneron.
Mirum CEO Chris Peetz stated that Atebrioz could be a game-changer for patients, as it combines oral dosing with strong efficacy and a favourable safety profile.
The company expects to launch the drug in October and will announce the price at that time.
About 300 people in the United States and 900 worldwide are living with FOP, according to Mirum.
