U.S. FDA approves Mirum Pharmaceuticals' zilurgisertib as a treatment for FOP. Posted on September 25, 2026 Atebrioz(TM) is the third treatment approved for people living with FOP in the United States. Today, September 25, is an incredible day for the FOP community! IFOPA want to start by thanking FOP community members from around the world who participated in the zilurgisertib trial. Mirum Pharmaceuticals and Incyte have announced the U.S. Food & Drug Administration's (FDA) approval of zilurgisertib tablets for the treatment of fibrodysplasia ossificans progressiva (FOP) in the United States. Zilurgisertib is the first FOP drug approved after completing only a Phase 2 clinical trial. * The brand name of the drug is Atebrioz(TM) * Once-daily oral ALK2 inhibitor approved to reduce the volume of total new heterotopic ossification in patients aged 12 years and older with FOP. * Mirum will soon release websites to help people with FOP and their local healthcare providers learn more about Atebrioz. * Atebrioz is expected to be available in the United States in October through Mirum Access Plus (MAP), with eligible patients paying as little as $0 per month. * Atebrioz is only available for treatment of FOP for individuals in the United States. * The European Medicines Agency (EMA) in the European Union is also reviewing zilurgisertib. Letter from Mirum's CEO. Thank you. Zilurgisertib (now known as Atebrioz) was tested in the PROGRESS Phase 2 clinical trial. IFOPA is deeply grateful to the 63 people with FOP who participated. IFOPA is also grateful to the Principal Investigators, Study Coordinators, and all of the staff at the trial sites who took such good care of its families. IFOPA want to thank Novartis, Incyte, and Mirum for their commitment and dedication to the FOP community. Publications & Presentations. The links below include press releases, a poster, and a plain language summary of the PROGRESS trial results. IFOPA expect a publication in a peer-reviewed journal. Presentations from the 2026 7th FOP Drug Development Forum Incyte shared this poster and plain-language summary of their results, which show an 81% reduction in the proportion of patients with new lesions at Week 24 (compared to placebo), at ENDO 2026, the leading global meeting on endocrinology research and clinical care hosted by the Endocrine Society. Where is Atebrioz approved? It is important to note that the FDA's approval of Atebrioz is only for people aged 12 and older with FOP living in the United States. On August 5, 2026, Mirum announced during an Investor Call that the EMA in the European Union accepted zilurgisertib for review. IFOPA await the EMA's decision. In what other countries is Mirum seeking approval of zilurgisertib? Zilurgisertib previously received Fast Track designation and Orphan Drug Designation from the FDA, as well as Orphan Designation by the European Medicines Agency in the European Union. IFOPA communicate regularly with Mirum about developments with zilurgisertib and will share announcements with the FOP community when they are publicly available. Is zilurgisertib in a trial for children younger than 12? The PROGRESS Phase 2 trial includes an active Cohort 2 for children ages 6 to < 12 years of age that is fully enrolled. Cohort 3 for children ages 2 to < 12 years is recruiting. You can learn more at ifopa.org/progress If you would like to receive updates on clinical trials and drug reviews, please sign up for IFOPA's Clinical Trial Alerts at ifopa.org/clinical-trial-alerts About zilurgisertib. People with FOP harbor mutations in the ALK2 protein (also known as ACVR1) that drives excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development. Zilurgisertib (INCB000928) is an oral investigational drug designed to target and block this disease-causing mutant FOP protein hyperactivity. In preclinical studies, zilurgisertib demonstrated potency for the target kinase, selectivity, safety and strong suppression of heterotopic ossification (HO) in animal models.

Mirum Pharmaceuticals has appointed Dr Rob Myers as chief medical officer, effective 21 September 2026. Dr Myers will lead the company's global medical and clinical development functions. Dr Myers brings over 20 years of experience in drug development, with particular expertise in hepatology. He joins from OrsoBio, where he served as chief medical officer and head of development. Previously, he held senior leadership positions at Gilead Sciences, overseeing clinical development programmes across liver fibrosis, cholestatic liver diseases, and gastrointestinal inflammation. Dr Myers has published more than 200 peer-reviewed papers and has contributed to advancing understanding of primary sclerosing cholangitis. He holds an MD from Western University and a master's degree in epidemiology from the University of Calgary.

US FDA approves Regeneron's rare bone disorder drug. Regeneron's Pasatru has received US FDA approval for adults with FOP after clinical trials showed a significant reduction in abnormal bone formation. By Reuters August 20, 2026, 8:59:40 AM IST (Published) The US FDA on Wednesday (August 19) approved Regeneron's drug for a rare genetic disorder after it significantly reduced abnormal bone formation in certain soft tissues. New York-based Regeneron's shares rose 4%. The drug, garetosmab, branded as Pasatru, was approved to treat adults with fibrodysplasia ossificans progressiva (FOP) - a condition in which muscle, tendon and ligament tissue gradually turns into bone, leading to a "second skeleton" that causes progressive loss of mobility and reduced life expectancy. In a 56-week trial involving 63 participants, Pasatru reduced the development of new bone abnormalities by 94% in patients treated with a 3 mg per kg dose and by 90% in those given a 10 mg per kg dose, compared with a placebo. The drug works by blocking Activin A, a protein involved in triggering abnormal bone growth in patients with FOP. Susan Rhee, a member of Regeneron's clinical team, told Reuters last week that the company plans to start a trial for children later this year. The drug will compete with French drugmaker Ipsen's oral treatment Sohonos, which in 2023 became the only other treatment approved by the US Food and Drug Administration. Incyte and partner Mirum Pharma, as well as privately held Ashibio, are also developing treatments for the condition. In 2020, Regeneron paused dosing in a mid-stage trial of the drug after five patient deaths, ultimately discontinuing the study and working with global regulators to design the late-stage trial, according to the company. The condition affects roughly 1 in 2 million people worldwide, with around 800 to 900 active diagnosed cases globally, according to data from the National Institutes of Health.