What Is Atebrioz, and How Does It Work?
The FDA has approved Atebrioz (zilurgisertib) for adults and children aged 12 or older who have fibrodysplasia ossificans progressiva (FOP). FOP is a very rare genetic disease that causes extra bone to form outside of the normal skeleton. Over time, soft tissues such as muscles, tendons, and ligaments gradually turn into bone. This extra bone limits body movement, causes deformities, and leads to severe disability.
FOP is caused by an abnormal gene that keeps a bone-growth protein turned on. Atebrioz works by blocking this active protein signal, which helps lower the amount of new abnormal bone that forms.
Why Was Atebrioz Approved?
The FDA approved Atebrioz based on a study of 63 adults and children aged 12 or older with FOP. Patients were randomly assigned to take either Atebrioz tablets once a day or placebo tablets (containing no real medicine). Researchers used whole-body CT scans to measure the volume of new bone growth after 24 weeks.
In the study, patients who took Atebrioz had an average decrease of 3.2 cubic centimeters in new extra bone volume. In contrast, patients who took the placebo had an average increase of 24.6 cubic centimeters in new extra bone volume.
What Do I Need to Know About Atebrioz?
Atebrioz is a tablet you take by mouth once a day, with or without food. You can swallow the tablets whole or crush them. If crushed, mix four tablets with four tablespoons of water, orange juice, applesauce, or plain yogurt, and take the mixture right away. Do not take Atebrioz with grapefruit, pomelo, or juices made from these fruits.
Atebrioz can cause harm to a fetus and must not be taken during pregnancy. If you can get pregnant, you must take a pregnancy test before starting Atebrioz. You must also use effective birth control during treatment and for one week after your last dose. If you become pregnant while taking Atebrioz, stop taking the medicine right away and call your healthcare provider. Breastfeeding is also not recommended during treatment and for one week after your last dose.
The most common side effects of Atebrioz include headaches, joint pain, nosebleeds, nausea, and upper respiratory tract infections such as colds. This medicine can interact with several other drugs, including strong CYP3A4 inhibitors and diabetes medicines such as metformin. Be sure to tell your healthcare provider about all the prescription drugs, over-the-counter medicines, and vitamins you take.