US FDA approves Mirum’s drug for rare bone disorder

Mirum Pharmaceuticals (MIRM) and Incyte (INCY) announced FDA approval of Atebrioz (zilurgisertib) for treating fibrodysplasia ossificans progressiva (FOP) in patients aged 12+. The once-daily oral ALK2 inhibitor showed efficacy in reducing heterotopic ossification in clinical trials. Atebrioz is expected to be available in the U.S. in October, with eligible patients paying as little as $0 per month through Mirum's patient support program.

The FDA approved Mirum Pharmaceuticals' drug Atebrioz for treating fibrodysplasia ossificans progressiva, a rare bone disorder. The once-daily oral treatment targets patients aged 12 and older, blocking the ALK2 protein to prevent abnormal bone formation. Mirum Pharmaceuticals is traded on the NASDAQ under the ticker MIRM.

Merck (MRK) awaits FDA decision on Winrevair for pulmonary arterial hypertension. Mirum (MIRM) faces decision on Zilurgisertib for FOP, with Incyte (INCY) holding economic interest. Grail (GRAL) has FDA advisory committee review for Galleri multi-cancer blood test. MRK up 40%, INCY up 23%, MIRM up 17.6%, GRAL down 9%.

Mirum Pharmaceuticals, Inc. (MIRM) filed an SEC Form 8-K — Results of Operations and Financial Condition. Exhibit 99.1 Mirum Pharmaceuticals Reports Second Quarter 2026 Financial Results and Provides Business Update – Q2 2026 net product sales of $176 million – 2026 net product sales guidance increased to $680 million to $700 million – Volixibat granted Breakthrough Therapy and Orpha

Mirum Pharmaceuticals reported Phase 2b results at the EASL Congress. In VISTAS, volixibat in primary sclerosing cholangitis reduced pruritus and the company plans an NDA submission in H2 2026. In AZURE-1, brelovitug in chronic hepatitis delta virus showed virologic response and ALT normalization at Week 24; Phase 3 topline data are expected later in 2026.

The article is largely promotional and does not provide verifiable details of Mirum Pharmaceuticals’ Phase 2b results for volixibat in PSC. It references an “EASL Congress” update but includes no specific efficacy/safety figures. It also promotes subscription offers and claims about prior stock-pick performance and “upside potential,” without citing sources.