Director Medical Safety, Global Patient Safety

Overview

Ready to join a team committed to moving gene therapies into the clinical setting 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 at the beginning of an incredible journey, and are looking for passionate individuals to join us on this important mission.

AveXis, Inc. a Novartis company, dedicated to developing and commercializing novel gene therapies 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.

The Director Medical Safety, a drug safety physician responsible for clinical safety and risk management for assigned products as well as medical review of individual adverse event reports, generation and review of aggregate reports and management of safety signals for assigned product(s) in clinical development and post-marketing.

This individual should be a self-starter with drug safety expertise in early and late stage clinical development program (e.g. gene therapy, biologics) who is seeking to be part of an innovative and fast-paced team to support the company's drug development activities.

Responsibilities

• Responsible for review of 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.
• Review of individual case safety reports originating from clinical trials and post-marketing.
• Responsible for conducting safety signal detection activities, monitoring, evaluation, interpretation, management and communication.
• Chair the cross-functional Safety Management Team for assigned products.
• Collaborate with clinical team, vendor and CRO safety physicians, medical monitors, toxicology group, and other functional groups in identification, analysis, and reporting of possible safety signals.
• Contribute to development & maintenance of product safety profile. Provide medical safety input into all critical documents (e.g. protocols and amendments, IBs, CSRs, submission dossiers, INDs, BLAs etc.).
• Track events of special interest and assist in development & maintenance of standardized queries for events of special interest.
• Develop strategy for the analysis in support of responses to regulatory, DSMB, IRBs and Investigators or ad hoc inquiries regarding safety issues.
• Develop 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. is required with 3-5 years of clinical practice experience in relevant areas (e.g. pediatrics, neurology).
• Min 5 years' 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 processes' improvements.
• Occasional travel (up to 10%) for conferences, meetings and professional development activities

Non-Essential Functions:

• Strives to maintain a safe working environment through the prevention of accidents, the preservation of equipment, and the achievement of safe working practices.
• Maintains a positive and professional demeanor toward all customers and coworkers.
• Adheres to all policies and procedures of AveXis.
• Performs other duties as assigned.

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.

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