Director, Medical Safety, Global Patient Safety

Overview

Ready to join a team committed to moving gene therapies into the clinical and commercial settings for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science to treat rare and life-threatening genetic diseases starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101. We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, Inc., a Novartis Company , is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality, and for the treatment of SMA Type 2. The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

AveXis is seeking a Director, Medical Safety to act as a drug safety medical expert responsible for clinical safety and risk management for assigned products as well as medical assessment of individual adverse event reports, generation and review of aggregate reports and management of safety signals from clinical program and post-marketing. The Director Medical Safety will lead the safety management team for assigned products and be responsible for the assessment of clinical safety and benefit-risk profiles across the product life cycle.

Must be a self-starter with drug safety expertise in early and late stage clinical development programs looking to be part of an innovative and fast-paced team supporting the company's drug development activities.

Responsibilities

• Review all safety data from various sources (e.g. pre-clinical, clinical trial data, post-marketing, literature) throughout the development process as well as post-marketing for assigned products.
• Conduct safety signal detection activities, monitoring, evaluation, interpretation, management and communication.
• Chair the cross-functional Safety Management Team for assigned products.
• Collaborate with the clinical team, vendor and CRO safety physicians, medical monitors, toxicology group, and other functional groups in the identification, analysis, and reporting of possible safety signals.
• Contribute to the development and maintenance of the product safety profile. Provide medical safety input into all critical documents (e.g. protocols and amendments, IBs, CSRs, submission dossiers, INDs, BLAs).
• Track events of special interest and assist in development and maintenance of standardized queries for events of special interest.
• Develop the strategy for analysis in support of responses to regulatory, DSMB, IRBs and Investigators or ad hoc inquiries regarding safety issues.
• Develop the strategy and analysis plan for periodic reports.
• Draft regulatory inquiry responses and provide oversight on key content of risk management documents (RMPs, REMs) and aggregate reports.
• Participate in regulatory meetings, interactions and submission activities as necessary.

Qualifications

• M.D. with 3-5 years of clinical experience.
• Clinical experience in relevant areas (e.g. pediatrics, neurology) desired.
• Minimum 5 years of experience in the pharmaceutical industry, predominately in drug safety, having worked on both clinical development and marketed products.
• Clinical development and/or drug safety experience with biologics, gene therapy products desirable.
• Credible knowledge of global regulations governing Pharmacovigilance for products in clinical development and post-marketing.
• Effective multi-tasking skills applied in different and complex assignments and ability to work effectively under pressure and under strict timelines.
• Ability to work both independently and collaboratively.
• Approachable, flexible and self-motivated..
• Open to changes, suggestions and innovations, continuously on the look-out for work process improvements.
• Occasional travel (up to 15%) for conferences, meetings and professional development activities.

The level of this position will be based on the final candidate's qualifications.

Please note this job description is not designed to cover or contain a comprehensive listing of activities, duties or responsibilities that are required of the employee for this job. Duties, responsibilities and activities may change at any time with or without notice.

AveXis is committed to creating a diverse environment and is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation or veteran status.