05/12/2026
This is exciting news for the FOP community! 💙💚
Mirum Pharmaceuticals announced that it has acquired the rights to zilurgisertib, a drug currently being studied as a possible treatment for FOP in the PROGRESS Phase 2 trial.
Even more encouraging, results from Part 1 of the trial (patients ages 12+) have been submitted to the FDA and granted Priority Review (meaning the review process will move faster than normal). The FDA is expected to make a decision by September 26, 2026.
The Scoble family is beyond grateful for your support for the past 19 years. A large portion of the funds donated to Joshua’s Future Of Promises have helped us reach this research milestone. Your donations are making a difference. Thank you to everyone who has supported our cause. 💙💚
For more information, visit ifopa.org.
We’re expanding our commitment to the rare disease community.
Mirum has in-licensed exclusive worldwide rights to zilurgisertib, an investigational oral therapy in development for fibrodysplasia ossificans progressiva (FOP)—an ultra-rare genetic disease, from Incyte.
The FDA has accepted the NDA for zilurgisertib and granted Priority Review, with a PDUFA date of September 26, 2026.
This moment reflects years of progress driven by the FOP community, and we’re honored to add our voice to that foundation.
For patients living with FOP and their families, new treatment options cannot come soon enough. We’re committed to working with urgency to bring this potential therapy forward.
