Senior Medical Director, Early Clinical Development

BioMarin Pharmaceutical Inc.; Northern (KY); Hybrid; USD 284,000 - 391,000; Full time

Job summary

BioMarin Pharmaceutical Inc. seeks a Senior Medical Director to lead early clinical development, from first‑in‑human studies to demonstration of clinical proof of concept.

You will shape strategy, oversee Phase 1/2 trials, and coordinate cross‑functional teams with regulatory and governance bodies to drive value and patient impact. The role requires deep translational expertise, leadership across multiple assets, and close collaboration with Regulatory, Clinical Operations, Biomarkers, and

Qualifications

• Senior Medical Director with end-to-end early clinical development leadership.
• Experience leading Phase 1 and Phase 2 trials and regulatory submissions.
• Ability to interact with governance bodies, health authorities, and cross-functional teams.

Responsibilities

• Develop clinical development plans for full asset lifecycle.
• Serve as CDT lead for assets from research through POC.
• Provide leadership across multiple assets and allocate resources.
• Make decisions at governance points considering safety and efficacy.
• Lead contributions to pre-IND/IND regulatory submissions.
• Collaborate to integrate innovative trial designs and act as Medical Monitor when appropriate.
• Design natural history studies and biomarker strategies.
• Mentor Early Clinical Development colleagues.
• Support business development with clinical input.

Skills

Clinical leadership

CDTL

Medical monitor

Regulatory submissions

Education

MD/DO/PhD in related field

Tools

IND submissions

Biomarker strategy

Job description

BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions.

Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

About Worldwide Research and Development

From research and discovery to post-market clinical development, our R&D 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 rare diseases.

Overview

BioMarin’s Early Clinical Development (ECD) team is seeking an accomplishedSenior Medical Directorto serve as a senior clinical leader responsible for shaping and executing early clinical development strategies across one or more assets within the early pipeline. This role encompassesend‑to‑endclinical leadership fromfirst‑in‑humanstudies through demonstration of clinical proof of concept, with accountability for scientific rigor, strategic clarity, and developmentdecision‑makingduring thehighest‑riskphases of development.

Leveraging deep expertise in translational clinical research andearly‑phasetrial design, the Senior Medical Director will lead integrated clinical development planning, design and oversee highly informative Phase 1 and Phase 2 studies, and guide key investment and governance decisions based on emerging safety, pharmacology, biomarker, and efficacy data. The role partners closely with colleagues in Early Clinical Development, Biomarkers, Clinical Pharmacology, Preclinical Safety, Clinical Safety, Regulatory, and Clinical Operations to ensure BioMarin’s assets are optimally positioned to generatehigh‑valueinsights that inform downstream development and regulatory strategy.

In addition toasset‑levelleadership, the Senior Medical Director will contribute broaderportfolio‑levelclinical perspective across BioMarin’s early pipeline, support evaluation of new internal programs and external business developmentopportunities, andprovide mentorship and leadership within the ECD organization. This position is expected to interact regularly with senior governance bodies, health authorities, and external scientific experts, and torepresentBioMarin as a thought leader in early clinical development.

Key Responsibilities:

Clinical Strategy and Leadership

• Develop clinical development plans that serve as the strategic foundation for full development of assigned assets, with emphasis ondemonstratingproof of mechanism first‑in‑humanandfirst‑in‑patientstudies, andsubsequentdemonstration of clinical proof of concept.
• Serve as clinical development team leader(CDTL) for assets from the research phase through completion of human POC studies.
• Provide scientific and clinical leadership across multiple assets, balancing strategic priorities and resource allocation.
• Make recommendations at key development and governance decision points, incorporating safety, efficacy, and benefit‑riskconsiderations.

Clinical Development Execution

• Lead clinical contributions topre-IND, IND, and otherstage‑appropriateregulatory submissions.
• Collaborate with Research and Translational Science colleagues to integrate innovative experimental designs that inform clinical developmentstrategy and regulatory filings.Serve as MedicalMonitor on selected studies, asappropriate.

Translational and Biomarker Strategy

• Design and execute natural history studies to (1) deepen understanding of targeted patient populations and (2) enable development of endpoints that support efficient demonstration of POC and/or regulatory approval.
• Partner with Biomarker and Clinical Pharmacology colleagues to ensure robust plans for patient selection, pharmacodynamic assessments, response biomarkers, and potential approval endpoints.

Portfolio, Talent, and Organizational Development

• Provide matrixed leadership and mentorship to Early Clinical Development colleagues.
• May directly manage and develop more junior Medical Directors.
• Define capability needs for the ECD function and contribute to recruitment, training, and retention strategies.
• Establish and evolve standard operating procedures, decision frameworks, and best practices across the ECD organization.

Business Development Support

• Serve as a clinical expert supporting new project identification, including internal pipeline opportunities and business development assessments.
• Translational and Biomarker Strategy
• Design and execute natural history studies to (1) deepen understanding of targeted patient populations and (2) enable development of endpoints that support efficient demonstration of POC and/or regulatory approval.
• Partner with Biomarker and Clinical Pharmacology colleagues to ensure robust plans for patient selection, pharmacodynamic assessments, response biomarkers, and potential approval endpoints.

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.

Equal Opportunity Employer/Veterans/Disabled

An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.

The salary range for this position is: $284,000 to $390,500. BioMarin considers a variety of factors when determining base compensation, including experience, qualifications, and geographic location. These considerations mean actual compensation will vary. This position may also be eligible for a discretionary annual bonus, discretionary stock-based long-term incentives (eligibility may vary based on role), paid time off, and a benefits package. Benefits include company-sponsored medical, dental, vision, and life insurance plans.

Equal Opportunity Employer/Veterans/Disabled

An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.

If you have a disability and believe you need a reasonable accommodation in order to search for a job opening or apply for a position, email [e-mail] with your request.
This email address is not for general employment inquiries or correspondence. We will only respond to those requests that are related to the accessibility of the online application system due to a disability.

BioMarin Pharmaceutical does not accept unsolicited resumes from recruiters or agencies and we will not be responsible for any fees related to resumes that are unsolicited. We handle all recruiting through our Talent Acquisition team – please do not contact hiring managers directly. Agencies who wish to offer their services to BioMarin Pharmaceutical may contact us at [e-mail] .
Applicants, please do not use this email address to submit resumes – if you do so, you will be redirected to apply through the website.