Executive Director, Global Medical Lead, Phenylketonuria [PKU]

Working from Home
Jul 28, 2021
Required Education
Position Type
Full time
BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options. BioMarin will continue to focus on advancing therapies that are the first or best of their kind.

BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development 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 rare diseases. Come join our team and make a meaningful impact on patients’ lives.


This position is responsible for providing global medical leadership to PKU GMAF Global Program across all related products, i.e. Kuvan, Palynziq and BMN 307(gene therapy). This individual has a global scope of responsibility in the Medical function and must align with colleagues across matrix/functions pro-actively.

This individual will interface externally with healthcare professionals, clinical investigators, regulatory agencies amongst others as well as with internal BioMarin departments providing medical insights and supporting the product development strategy. 

This person must be able to provide insightful guidance to BioMarin’s lead PKU development and show deep knowledge of the current status of the science as well as of agents in the pipeline and associated topics.  This position will be the scientific reference at the global level, representing the department and the company at key internal and external activities and events.  This person’s responsibility will also include providing input on other cross-functional activities and initiatives, from Market Planning to Government Affairs, Market Access, Regulatory, Clinical Sciences and other departments, aligning with colleagues across matrix/functions pro-actively. S/he will represent BioMarin at scientific, clinical and business development meetings.


  • Responsible for the development of PKU portfolio strategy (in the form of the Global Medical Plan) in conjunction with a variety of internal stakeholders across BioMarin. In addition to the development of the strategy, direct management of projects and matrix collaboration within and outside of the GMAF department will be a key element of the job.
  • Represent BioMarin GMAF at regulatory meetings (as required) and serve as Subject Matter Expert internally (and externally) to ensure appropriate representation of PKU’s risk benefit profile by the relevant regulatory bodies.
  • Responsible for providing medical input into development, execution and interpretation of clinical protocols.  This includes review of draft independent research protocols, reports and manuscripts. S/he will oversee clinical registries and other post-marketing commitments and will evaluate safety issues associated with PKU program in collaboration with other departments. S/he will assist in critically evaluating study results in 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. Direct medical writing activities associated with Medical Affairs to ensure accurate execution of product dossiers, product related slide decks, standard response letters, FAQs as well as corporate abstracts, posters, oral presentation and manuscripts when appropriate.
  • Responsible for direct interaction with investigators, KOLs and the research community to support company initiatives. Responsible for representing BioMarin at scientific, clinical and business development meetings. Promote effective communication between internal and external groups regarding Medical Affairs initiatives
  • Provides subject matter expertise as well as leadership in different internal committees ensuring the most appropriate scientific and clinical input is incorporated into the global strategy, serving as medical and technical resource for clinical issues raised by internal and external collaborators.
  • Responsible for development and/or medical input in support of key medical messaging such as core claims documents, monographs, publication plans, etc, including identification of data gaps and proactive resolution of those. S/he will contribute to promotional activities for BioMarin products being done in accordance within the respective regulatory and compliance requirements.
  • Ensure compliance with policies, SOPs, Code of Business Conduct and Corporate Social Responsibility, as well as relevant legislation and regulations


  • Ability to work proactively and effectively, with exceptional creative problem-solving skills and self-motivation.
  • Excellent project management skills, with a drive for results coupled with the highest medical and ethical standards.
  • Ability to promote effective communication between internal and external groups. Collaborative and flexible in personal interactions at all levels of the company.
  • Able to thrive in an ambiguous and demanding environment, with high capacity for effective relationship building and teamwork.
  • Flexibility and adaptability. Sensitivity to a multicultural environment.
  • Excellent written and oral communication skills. Able to convince, inspire and motivate people with a variety of backgrounds
  • Solid business acumen with high scientific inquisitiveness.
  • Willingness to travel approx. 60% of the time, mostly internationally


Advanced Degree (M.D. / Ph.D., or both) in Medicine and/or a medical sciences field. Current Medical Registration / Revalidation desirable, but not required. Experience in PKU is highly desirable, either in a pharmaceutical/clinical research or a clinical environment. Experience in Genetic Medicine is a plus. 

Very strong Medical Affairs experience required.

English and one other language preferred.


  • Strong industry experience, especially in Medical Affairs (≥ 7 years).
  • Knowledgeable and current in GCP guidelines and compliance rules globally. Clinical trial and publication experience is desirable.
  • Demonstrate excellent medical affairs skills and ideally have working knowledge in the therapy area.
  • Willingness to travel mostly international and s/he will be required to interact closely with GMAF and other colleagues in San Rafael, CA.


Global Medical Affairs (GMAF) is the scientific and medical function that develops and maintains non-promotional, evidence-based, transparent and balanced scientific and educational programmes and resources in support of BioMarin’s products and disease states of interest.  We do this by assuring safe and appropriate product use, support of continuous scientific exchange and through ongoing and new data generation.

Within BioMarin Medical Affairs, the Medical Sciences group is responsible for being the subject matter experts on product scientific and medical knowledge, setting the Medical Affairs product strategy and leading global Medical Affairs activities for each BioMarin product progressing from pipeline status through product launch to post approval status. This includes collaborating with cross-functional stakeholders to define product strategy, developing and communicating key scientific messages to stakeholders in preparation for a commercial product launch; leading efforts to develop additional scientific evidence to support commercial product viability; and acting as the critical link between BioMarin and the external health care practitioners by supporting scientific and medical knowledge exchange.

Note:  This description is not intended to be all-inclusive or a limitation of the duties of the position.  It is intended to describe the general nature of the job that may include other duties as assumed or assigned.

We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law.