Jobs and Careers

Associate Director, Lysosomal Storage Disorder Programs

United States, United Statesfull_timeVerifiedPosted 23 Sept 2024

About the role

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.

Title: Medical Director, Lysosomal Storage Disorder (LSD) Programs, United StatesLocation: Remote Who We AreBioMarin 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.Global Medical Affairs (GMAF) is an expert scientific and medical function that develops and maintains non-promotional, evidence-based, transparent and balanced scientific and educational programs 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. The RoleThe US Lysosomal Storage Disorder (LSD) Medical Director is a critical strategic and execution focused role covering the following disease areas: MPS IVA (Vimizim), MPS VI (Naglazyme) and CLN2 (Brineura). The role reports into the US Medical Lead for PKU and LSDs and represents Medical Affairs on core cross-functional teams. Key ResponsibilitiesDisease Area Strategy & Execution

Develop and implement the US Medical Plan and lead US medical activities in coordination with Medical Affairs, Commercial and Regulatory colleagues
Ensure adherence to US regulatory and clinical practice guidelines in supporting development and marketing of BioMarin compound
Act as the US product and disease area expert and primary contact point for all compound or disease area related issues both from within Medical Affairs and all other BioMarin departments
Support internal and external communications related to diagnostic testing programs

Cross Functional Leadership

Represent Medical Affairs on relevant US Commercial teams including Brand Team

Represent Medical Affairs in the Promotional Review Board (PRB) and provide medical review of promotional material to ensure maximum effectiveness while adhering to the company's ethical standards and relevant Codes of Practice for the region
Provide medical oversight for Commercial international symposiums and advisory boards

Launch and Pre-Launch Excellence

Develop US launch plans in collaboration with Medical Affairs and Commercial colleagues and execute on the US specific tactics in support of global product launches

Filing / Regulatory

Assist in preparation and review of relevant product BLAs or sBLAs and other regulatory documents including protocols, protocol amendments and briefing materials.

Post Marketing Commitments