Vice President, U.S. Medical Affairs & Patient Advocacy

Concord, MA

About This Role:

The Vice President, US Medical Affairs & Patient Advocacy will provide strategic, organizational, and scientific leadership for ITF Therapeutics' U.S. Medical Affairs and Patient Advocacy organizations. This executive will be responsible for developing and executing an integrated U.S. Medical & Patient Advocacy strategy across two distinct pediatric therapeutic areas: neuromuscular disease and neurometabolic disease.

The scope of the role includes: (a) leading and further developing a high-performing Medical Affairs and Patient Advocacy organization supporting the continued growth of DUVYZAT ® in Duchenne muscular dystrophy; (b) building and preparing a new functional team to support the anticipated launch of a novel therapy in pediatric neurometabolic disease; and (c) continuing to scale capabilities, systems, and talent to support the future growth of the company.

The Vice President will serve as a key member of the U.S. Executive Leadership Team and a trusted strategic partner to Global Medical Affairs, Research & Development, and Commercial leadership. The successful candidate will bring executive presence, strong business acumen, scientific and medical credibility, and a demonstrated ability to influence internal and external stakeholders. This leader will represent the voice of the U.S. market in global decision-making and help shape the long-term medical and scientific strategy of the organization, globally.

This leader will embody and foster ITF Therapeutics' core values while developing a culture of excellence, collaboration, and patient-centered innovation.

What You'll Do:

Strategic Leadership

• Lead the development and execution of integrated U.S. Medical Affairs & Patient Advocacy strategies aligned with corporate objectives.
• Serve as a strategic thought partner to the U.S. President, Executive Leadership Team, Global Medical Affairs leadership, and R&D leadership in shaping business and portfolio strategy.
• Represent U.S. Medical Affairs perspectives and market insights within global cross-functional teams, ensuring alignment of development, launch, and lifecycle strategies.
• Provide senior medical leadership for DUVYZAT ® and pipeline assets, integrating scientific, clinical, regulatory, market, and patient considerations.
• Lead, develop, and scale a high-performing U.S. Medical Affairs & Patient Advocacy organization supporting both current and future rare disease therapeutic products.
• Mature organizational capabilities, talent, and infrastructure to support launch readiness, commercialization, and future portfolio growth.
• Develop leaders through coaching, succession planning, and performance management while fostering a culture of accountability, collaboration, and continuous improvement
• Serve as a member of the Global Medical Governance Team to (a) ensure US medical insights and business needs inform Global strategy and decision-making and (b) foster aligned, interdependent US and Global operating frameworks, processes, systems, and structures that enable efficient and seamless execution

Scientific & Medical Leadership

• Serve as the senior medical leader for the U.S. business, providing expert scientific guidance to cross-functional and executive stakeholders.
• Maintain strong scientific and medical credibility with key opinion leaders (KOLs), healthcare professionals, academic investigators, advocacy organizations, and professional societies.
• Develop and oversee a comprehensive KOL engagement and insights-generation strategy that informs business decisions and medical priorities.
• Drive scientific exchange, medical education, and non-promotional engagement activities that advance patient care and disease awareness.
• Collaborate with Global Medical peers to influence and execute evidence-generation strategies, including investigator-sponsored studies, real-world evidence initiatives, and other non-registrational research activities.
• Support late-stage clinical development and launch preparedness activities by providing strategic medical insight and ensuring alignment with evolving standards of care.

Operational Excellence & Governance

• Be accountable for the U.S. Medical Affairs & Patient Advocacy budgets and resource allocation to maximize organizational effectiveness and impact.
• Build and scale systems, capabilities, and processes to support a growing and increasingly complex medical…