Director, Global Medical Lead - Phenylketonuria (PKU)

Who We Are

BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health.

Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities.

Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options.

Our Culture

From research and discovery to post-market clinical development, our WWRD engine involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with genetic diseases.

• Leads development, execution, and communication of the Global Medical Plan in collaboration with other internal stakeholders across BioMarin
• Provides subject matter expertise as well as leadership in key internal medical and clinical teams, called SMART teams, where s/he leads development of the medical plan and ensures that all critical components of medical plans are executed and monitored for the intended impact
• Act as product and disease area expert and primary contact point for all global compound or disease area related issues both internally and for the external community.

• Develop Medical Affairs launch strategy and roadmap and lead all Medical Affairs launch activities in collaboration with Commercial and other key stakeholders

• Represent Medical Affairs in development of regulatory documents and submissions (e.g. briefing books, protocols, label discussions etc.)
• Represent Medical Affairs at FDA/EMA advisory board meetings

• Leads the Integrated Evidence Team for development of the integrated evidence package for the therapeutic area. Ensures timely execution of the plan to ensure data gaps are filled optimizing patient access.

• Responsible for providing medical input into development, execution and interpretation of Clinical Development Plans, clinical protocols, including review of draft external research protocols, reports and manuscripts
• Oversight of clinical registries and other post-marketing commitments
• Evaluation of safety issues associated with product in collaboration with other departments
• Assistance in critically evaluating study results for statistical and final reports and ongoing reviews with study investigators, resolving problems arising in the course of clinical investigations and preparing reports to management and medical community.

• Guide development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, tools are developed to support execution, and mechanisms are in place to measure impact  
• Contribute to the development of, review and approval of relevant global publication plans. Ensure publications are appropriately shared with the external community to optimize patient care.

• Support internal dis