ATEBRIOZ (Zilurgisertib)

ATEBRIOZ (zilurgisertib) is an oral medicine approved by the US FDA on 25 September 2026 to reduce the volume of total new heterotopic ossification (HO) in adults and children aged 12 years and older with fibrodysplasia ossificans progressiva (FOP). In FOP, muscles, tendons and ligaments gradually turn into bone, which locks joints and limits movement over time.

FOP is caused by a gain-of-function change in the ACVR1 gene, which codes for the ALK2 receptor. The faulty receptor keeps bone-forming signals switched on. Zilurgisertib selectively inhibits both the normal and the R206H mutant form of ALK2, which reduces this abnormal bone formation.

Approval was based on the 24-week placebo-controlled part of the PROGRESS study in 63 patients (28 adults and 35 adolescents aged 12 to 16). On whole-body CT scans, total new HO volume changed by an average of −3.2 cm³ with zilurgisertib compared with +24.6 cm³ with placebo. The FDA reviewed it under priority review as a new molecular entity with orphan drug status.

It is a once-daily tablet that can also be crushed and mixed with water, orange juice, apple sauce or plain yogurt for patients who find swallowing difficult. Families with questions about rare-disease imports can also read our frequently asked questions.

• Must not be used in pregnancy. Animal studies showed harm to the unborn baby, including skeletal malformations. A pregnancy test is needed before starting, effective contraception must be used during treatment and for 1 week after the last dose, and the medicine is stopped at once if pregnancy occurs. Breastfeeding is not recommended during treatment or for at least 1 week afterwards.
• Strong CYP3A4 inhibitors (for example itraconazole) roughly doubled zilurgisertib levels, so the dose is reduced to 50 mg a day with them. Strong or moderate CYP3A4 inducers such as rifampin lowered levels by about 79% and should be avoided.
• Zilurgisertib blocks the MATE1 and MATE2-K kidney transporters, so it should not be combined with medicines that rely on them, such as metformin. Grapefruit, pomelo and their juices should also be avoided.
• Side effects seen more often than with placebo in the first 24 weeks were headache (22% vs 16%), joint pain (22% vs 10%), upper respiratory infection (22% vs 10%), nosebleeds (13% vs 0%) and nausea (13% vs 3%).
• Average serum iron, haemoglobin and transferrin saturation rose but stayed in the normal range. Rat studies found liver changes whose relevance to people is not known, so regular review by the treating team is sensible.
• Safety and effectiveness have not been established in children younger than 12 years.

Who can be prescribed ATEBRIOZ?

Patients aged 12 years and above with a confirmed diagnosis of FOP, under the care of a specialist who manages the condition, usually an endocrinologist, geneticist or bone-disease specialist.

What papers are needed to import it for a patient?

• Prescription from the treating specialist with the daily dose
• Diagnosis summary, ideally with the ACVR1 genetic test report
• For female patients of child-bearing age, the doctor’s note that pregnancy has been ruled out
• Patient’s photo ID and delivery address

How long does one bottle last?

The usual dose is 100 mg once a day, which is four 25 mg tablets. One bottle of 120 tablets is therefore about a 30-day supply. If the dose is lowered to 50 mg because of an interacting medicine, the same bottle lasts about 60 days.

How should it be stored?

At room temperature (20–25 °C, short excursions between 15 and 30 °C are allowed), in the original bottle. The FDA approval letter gives a shelf life of 24 months from manufacture, so no cold chain is needed.

Is ATEBRIOZ sold in India?

No. As of October 2026 it is approved only in the USA and has not been approved by CDSCO. It can be imported for an individual patient on a specialist’s prescription once supply from the US distributor is confirmed.

ATEBRIOZ is distributed in the USA by Mirum Pharmaceuticals. We source it only through authorised channels for a named patient, confirm the expected delivery time and price in writing, and dispatch only after the prescription has been reviewed.

For the ATEBRIOZ price in India and current lead time: Call +91 96548 60915, WhatsApp +91 93100 90915 or email info@indianpharmanetwork.in.

Global Approval Status:

• United States: FDA approval on 25 September 2026 (NDA 221198; new molecular entity, priority review, orphan drug, rare pediatric disease priority review voucher).
• European Union: Orphan designation only (EU/3/25/3119); no marketing authorisation as of October 2026.
• India: Not approved by CDSCO.

Sources:

• ATEBRIOZ US Prescribing Information and Medication Guide (FDA, September 2026)
• FDA approval letter, NDA 221198
• Drugs@FDA record for NDA 221198
• EMA orphan designation EU/3/25/3119

Information last checked: October 2026.

What is ATEBRIOZ used for?

To reduce the amount of new abnormal bone that forms in people aged 12 and over who have fibrodysplasia ossificans progressiva (FOP).

How does zilurgisertib work?

It blocks ALK2, the overactive receptor behind FOP, which reduces the signals that turn soft tissue into bone.

Can the tablets be crushed?

Yes. Four tablets can be crushed and mixed with 60 mL of water, orange juice, apple sauce or plain yogurt and taken straight away.

Can ATEBRIOZ be taken with metformin?

It is not recommended, because zilurgisertib blocks the kidney transporters that clear metformin. Tell the doctor about every medicine being taken.

Who makes ATEBRIOZ?

It is marketed in the USA by Mirum Pharmaceuticals under a licence from Incyte Corporation, which owns the ATEBRIOZ trademark.

Is ATEBRIOZ FDA approved?

Yes, since 25 September 2026, for adults and children aged 12 years and older with FOP.