Mirum raises 2026 sales guidance, volixibat gets FDA breakthrough status for PSC

Mirum Pharmaceuticals reported second quarter 2026 results on August 5, 2026, and raised its full-year net product sales guidance to $680 million to $700 million.¹

On the volixibat program in cholestatic pruritus due to primary sclerosing cholangitis (PSC), the FDA granted volixibat Breakthrough Therapy Designation and Orphan Drug Designation for PSC.¹ CEO Chris Peetz said the VISTAS study was designed with FDA input as a pivotal study and met its primary endpoint with highly significant results.¹ However, he said at the company's recent pre-NDA meeting, the agency recommended conducting a Phase 3 study.¹ Mirum said it plans additional discussions with the FDA before potential NDA submission, now targeted for the first half of 2027.¹

In a separate volixibat program, the company said it had finished enrolling patients in the VANTAGE Phase 2b trial for cholestatic pruritus tied to primary biliary cholangitis, with topline data expected in the first quarter of 2027.¹

Other pipeline updates: topline results from the brelovitug AZURE-1 and AZURE-4 Phase 3 studies in chronic hepatitis delta virus are expected in the third and fourth quarters of 2026, respectively.¹ Topline results from the LIVMARLI EXPAND Phase 3 study in additional rare cholestatic conditions are expected in the fourth quarter of 2026.¹ For zilurgisertib in fibrodysplasia ossificans progressiva, the company presented positive pivotal Phase 2 PROGRESS study results at ENDO 2026, with an FDA target action date of September 26, 2026 under the Prescription Drug User Fee Act.¹

On finances, Mirum had unrestricted cash, cash equivalents, and investments of $561.3 million as of June 30, 2026, compared with $391.4 million at the end of 2025.¹ The company also issued $690.0 million in 0.00% convertible senior notes due 2032 and settled $237.2 million of its 4.00% convertible notes due 2029, about 75% of those then outstanding.¹