The US Food and Drug Administration (FDA) has approved a new drug, zilurgisertib, for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare and severe genetic bone disorder.
Developed by Mirum Pharmaceuticals, the drug will be marketed in the US under the brand name Atebrioz. According to the company, the once-daily treatment has been approved for patients aged 12 and older who have FOP.
FOP is a rare genetic disorder in which muscles, tendons and ligaments gradually turn into bone, progressively restricting movement and potentially causing severe disability.
According to a Reuters report, FOP is usually diagnosed during early childhood. Mirum Pharmaceuticals estimates that around 300 people in the US and approximately 900 worldwide are affected by the condition.
Zilurgisertib works by blocking a protein known as activin receptor-like kinase 2 (ALK2). The protein is abnormally active in most FOP patients and plays a key role in the formation of bone outside the normal skeleton. The recommended dose is 100 milligrams once a day.
The FDA approval was based on results from a clinical trial involving 63 patients. After 24 weeks, patients receiving the drug showed a significant reduction in abnormal bone formation compared with those given a placebo.
Mirum Pharmaceuticals Chief Executive Chris Peetz said the company plans to launch the drug in October, with its price expected to be announced around the same time.
Other treatments are also available for FOP, including Ipsen’s oral drug Sohonos and Regeneron’s monthly infusion Pasatru. Sohonos is approved for certain children and adults, while Pasatru is approved for adult patients.
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