Global Medical Lead LC-FAOD

Why Join Us?   Be a hero for our rare disease patients   At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them.    Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work.   If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team.

Position Summary:

Ultragenyx is seeking a strategic and visionary physician leader to join the Global Medical Affairs team focused on LC-FAOD and triheptanoin.  

Reporting to the VP Global/US Medical Affairs and Strategy, the Global Medical Lead will be an adaptable leader, willing to take a “hands on” approach and comfortable working in a fast-paced intellectual environment.  This leader will be adept at leveraging their inborn errors of metabolism expertise to advance our mission to change the future of rare disease medicine. 

ultrainnovative – Tackle rare and dynamic challenges

Work Model: 

Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.

Responsibilities:

• Lead the Global Medical Sub Team (GMST), ensuring regional medical affairs are involved in and inform the development of global medical strategy 

• Develop and drive the timely execution of the Global Medical Plan for LC-FAOD and triheptanoin in the pre-launch, launch, and post-launch phases  

• Serve as the strategic medical leader on the Program Core Team and key sub-teams, shaping development, commercialization, and lifecycle strategies through deep scientific and clinical expertise. Work decisively to overcome challenges and drive program priorities forward 

• Lead advisory boards, steering committees, and investigator meetings to gather key insights that inform program strategy and activities 

• Lead updates to the Integrated Evidence Generation Plan (IEP), ensuring alignment with HEOR and clinical development to address global/regional evidence gaps 

• Lead and successfully complete global medical affairs data generation activities, including real-world evidence (RWE), investigator-sponsored trials (ISTs), and other strategic evidence initiatives  

• Accountable as the medical affairs lead for Disease Monitoring Programs for LC-FAOD, providing strategic medical leadership on all aspects of the study and accountable for leading data generation and publication activities aligned to the IEP 

• Provide deep disease area and product expertise, ensuring accurate and consistent communication of clinical and scientific data 

• Lead global publication strategy and congress planning activities, ensuring timely, high-impact disseminatio