Global Medical Director LC FAOD and MPS

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.

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. 

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. 

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