Senior Medical Director, Early Clinical Development

BioMarin Pharmaceutical Inc.

71d

About The Position

BioMarin’s Early Clinical Development (ECD) team is seeking an accomplished Senior Medical Director to 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 encompasses end‑to‑end clinical leadership from first‑in‑human studies through demonstration of clinical proof of concept, with accountability for scientific rigor, strategic clarity, and development decision‑making during the highest‑risk phases of development. Leveraging deep expertise in translational clinical research and early‑phase trial 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 generate high‑value insights that inform downstream development and regulatory strategy. In addition to asset‑level leadership, the Senior Medical Director will contribute broader portfolio‑level clinical perspective across BioMarin’s early pipeline, support evaluation of new internal programs and external business development opportunities, and provide 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 to represent BioMarin as a thought leader in early clinical development.

Requirements

Accomplished Senior Medical Director
Senior clinical leader
Deep expertise in translational clinical research and early-phase trial design
Experience leading integrated clinical development planning
Experience designing and overseeing Phase 1 and Phase 2 studies
Experience guiding investment and governance decisions based on emerging data
Experience partnering with cross-functional teams (Early Clinical Development, Biomarkers, Clinical Pharmacology, Preclinical Safety, Clinical Safety, Regulatory, Clinical Operations)
Experience contributing portfolio-level clinical perspective
Experience supporting evaluation of new internal programs and external business development opportunities
Experience providing mentorship and leadership
Experience interacting with senior governance bodies, health authorities, and external scientific experts
Experience representing a company as a thought leader in early clinical development
Experience leading clinical contributions to regulatory submissions (pre-IND, IND, etc.)
Experience designing and executing natural history studies
Experience partnering with Biomarker and Clinical Pharmacology colleagues on patient selection, pharmacodynamic assessments, response biomarkers, and potential approval endpoints

Nice To Haves

Experience in rare diseases
Experience with specific therapeutic areas such as achondroplasia, PKU, CLN2, MPS, Fabry disease, or Pompe disease

Responsibilities

Develop clinical development plans that serve as the strategic foundation for full development of assigned assets, with emphasis on demonstrating proof of mechanism first‑in‑human and first‑in‑patient studies, and subsequent demonstration 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‑risk considerations.
Lead clinical contributions to pre-IND, IND, and other stage‑appropriate regulatory submissions.
Collaborate with Research and Translational Science colleagues to integrate innovative experimental designs that inform clinical development strategy and regulatory filings.
Serve as Medical Monitor on selected studies, as appropriate.
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.
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.
Serve as a clinical expert supporting new project identification, including internal pipeline opportunities and business development assessments.