About this role
Be a part of a global team inspired to make a difference in the lives of people living with rare disease. The Director, Global Medical Affairs – Rare Metabolic Disorders serves as a strategic scientific leader supporting a Phase 3 asset in the genetic metabolic disease space. This role shapes and executes global medical strategy in preparation for potential regulatory approval and launch.
Act as a subject matter expert in inborn errors of metabolism, leading evidence generation planning, scientific narrative development, and external engagement with professional societies. Translate Phase 3 data into a compelling and differentiated scientific story through close collaboration with Clinical Development. Work cross-functionally with Commercial, Market Access, Medical Training, Scientific Communication, and Field Medical teams.
Thrive in a collaborative, diverse, fast-paced environment while sharing the mission to identify, develop, and deliver life-changing therapies to rare patients. Stick by values centered on patients, courage, community, and collaboration. Establish strong external partnerships that advance disease understanding and patient care.
Pursue the vision of becoming a leading biopharmaceutical company dedicated to innovation and hope in the global rare disease community. Forge new paths elevating science and service for rare patients. Experience rewarding work both professionally and personally by making a difference.
Requirements
- Subject matter expertise in inborn errors of metabolism
- Strong foundation in rare metabolic disorders
- Experience shaping global medical strategy for Phase 3 assets in genetic metabolic diseases
- Proven ability to lead evidence generation planning and data interpretation
- Track record in scientific narrative development and external stakeholder engagement
- Cross-functional collaboration with Clinical Development, Commercial, and Market Access teams
- Knowledge of professional societies, guideline bodies, and key opinion leaders in rare diseases
- Strategic experience in pre-launch medical affairs for rare disease therapies
Responsibilities
- Support overall Medical strategy for rare metabolic conditions
- Translate Phase 1/2 and Phase 3 data into clear scientific insights and positioning
- Provide strategic input across initiatives, ensuring scientific rigor and clinical relevance
- Serve as the internal medical expert in rare metabolic disorders with strong foundation in inborn errors of metabolism
- Lead integrated evidence generation planning aligned with Phase 3 and pre-launch needs
- Identify and prioritize evidence gaps critical for regulatory, payer, and clinical adoption
- Develop and execute a strategic engagement plan with US professional societies and scientific organizations
- Represent the company at advisory boards, congresses, and scientific forums
Benefits
- Remote work option from San Diego, USA
- Collaborative, diverse, fast-paced environment
- Mission-driven focus on rare patients and life-changing therapies
- Rewarding work making a professional and personal difference
Similar roles

Senior Medical Director - Drug Safety & Pharmacovigilance
1w1 week agoScholar Rock
Cambridge, US · Full-time · $275,000 – $375,000

Medical Writer (Remote)
1w1 week agoStarfish
IN · Full-time · $95,000 – $135,000

Medical Science Liaison (MSL)
1w1 week agoChiesi Group
Brussels, BE · Full-time · €70,000 – €95,000

Senior Medical Affairs Scientific Director
1w1 week agoGrifols
US · Full-time · $200,000 – $275,000
