Global Medical Director LC FAOD and MPS

Ultragenyx Pharmaceutical

30d•Remote

About The Position

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: ultrafocused – Work together to fearlessly uncover new possibilities Ultragenyx is seeking an experienced physician leader with a passion for rare and ultra-rare diseases. Reporting to the Global Medical Lead, the Director will be a key contributor to shaping the strategy and executing the medical affairs plans for LC-FAOD, MPS VII and MPS IIIA. This role offers a unique opportunity to be part of highly collaborative cross-functional teams working on both approved therapies (Dojolvi and Mepsevii), and an investigational AAV gene therapy (UX111) as we prepare for future potential launches. Work Model: Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.

Requirements

Medical Doctor (MD or DO) required.
Clinical expertise in the management of patients with inherited metabolic and/or neurodegenerative rare diseases strongly preferred.
At least 2 years of experience in the pharmaceutical industry is preferred, but extensive relevant clinical experience may be considered as an alternative.
Strategic thinker with strong execution discipline and a proven ability to manage multiple projects.
Ability to interpret and communicate complex clinical and scientific information to a range of audiences.
Highly analytical in approach to work, with the ability to identify interdependencies, consider the medical implications and understand the broader goals of a program and the organization.
Ability to work independently and as a member of a cross-functional matrix team in a fast-paced environment.
High attention to detail, demand for high-quality work, and sense of passion and urgency to achieve goals and improve the lives of patients.
Willingness and ability to travel up to 15%, including domestic and international engagements.

Responsibilities

Provide strategic medical and scientific leadership to LC‑FAOD (Dojolvi) and the MPS programs (MPS VII and MPS IIIA) to support the development and execution of the global medical plans.
Contribute to the development and execution of the annual Integrated Evidence Generation Plans.
Provide medical expertise and leadership to Real‑World Evidence initiatives (e.g., ISTs, collaborative research) and the Disease Monitoring Programs for LC‑FAOD and MPS focused on data generation and publication activities.
Act as a subject matter expert in assigned therapeutic areas, guiding and contributing to the execution of key global medical affairs activities including advisory boards, medical education initiatives and congress activities.
Lead robust scientific engagement and education, maintaining strong relationships with external stakeholders to generate insights and advance scientific understanding of assigned programs.
Provide scientific and medical leadership as needed for publication and medical communication material development.
Serve as a medical reviewer for promotional materials and non-promotional scientific materials ensuring accuracy, balance, strategic alignment and compliance with regulatory requirements.
Provide subject matter expertise to develop and deliver training across the organization.
Collaborate cross‑functionally with Clinical Development, Commercial, Regulatory, HEOR, Biostatistics, Patient Advocacy, Regional Medical Teams and others to achieve program goals.