To learn more about Mirum Pharmaceuticals' recent announcement regarding zilurgisertib, read the full press release and community letter here.
These Frequently Asked Questions were developed with input from Mirum Pharmaceuticals and Incyte Corporation.
1. Who is Mirum Pharmaceuticals?
Mirum Pharmaceuticals (Mirum) is a rare disease company dedicated to developing treatments for people living with rare and ultra-rare diseases. Mirum focuses on areas where the burden on patients and families can be significant and where treatment options may be limited. The company works closely with patients, families, patient advocacy groups, and healthcare professionals to better understand these conditions and develop medicines that can make a meaningful difference in patients’ lives. Learn more at www.mirumpharma.com.
2. Where is Mirum based?
Mirum is headquartered in Foster City, California, with additional offices in North America and Europe and team members supporting patients, caregivers, patient advocacy groups, and healthcare providers around the world.
3. Does Mirum have rare disease experience?
Yes. Mirum is a rare disease company with deep experience developing and commercializing medicines for rare conditions, including rare liver and genetic diseases. The company has three approved medicines, ongoing research programs, and a growing global footprint and pipeline, reflecting its continued focus on rare diseases. Mirum combines scientific expertise with a strong commitment to understanding what individuals living with rare diseases and their families experience in their daily lives. Those insights help shape how Mirum approaches development and how it engages with the communities it serves.
4. How will Mirum learn about FOP and patients' needs?
Mirum will learn by listening closely to people living with FOP, their families, caregivers, healthcare providers, researchers, and advocacy groups such as IFOPA. Understanding lived experience is an important part of how Mirum approaches rare diseases, and those perspectives will help shape how the company engages with and supports the FOP community over time.
5. Why would Incyte enter into an agreement granting Mirum worldwide commercial rights to zilurgisertib?
Companies sometimes partner to bring potential new medicines forward with the right combination of focus, expertise, and infrastructure. In this case, Mirum and Incyte have structured the agreement to leverage the strengths of both organizations. Incyte will continue to lead the development of zilurgisertib through potential approval, while Mirum will assume commercialization responsibilities, if zilurgisertib is approved. Drawing on its rare disease expertise and global commercial capabilities, Mirum aims to deliver potential treatment options with urgency and purpose.
6. Will any patients finish the 292 weeks before September 26? If so, what happens? Do they lose access to the drug?
No. There are no planned changes to the conduct of the PROGRESS study as a result of this agreement including the open label extension period, and current study participants will not lose access to zilurgisertib. Mirum and Incyte understand continuity of care is critically important to the FOP community.
7. Will there be any changes to the regulatory path or clinical development of zilurgisertib or the conduct of the PROGRESS study at this time?
Incyte will continue to lead current development activities for zilurgisertib, including the ongoing PROGRESS study, and there are no planned changes to study conduct as a result of this agreement. Mirum and Incyte share a goal of advancing potential new treatment options for people living with FOP with urgency and purpose. We are not able to speculate on potential regulatory outcomes or hypothetical next steps, but meaningful updates will be shared with the FOP community as appropriate.
Have another question?
If you have additional questions that weren't addressed in these FAQs, please don't hesitate to contact us at [email protected].
