Regional Medical Director, US Medical Affairs-Southern California

AveXis Inc.
Oct 07, 2018
Required Education
Position Type
Full time

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.

The Regional Medical Director will support US Medical Affairs in order to execute the consolidated US Medical Affairs strategy; ensure accurate, robust and appropriate medical/scientific exchange of knowledge and clinical expertise with US stakeholders for AVXS-101; and partner with internal stakeholders in driving success.


  • Executes the company's US Field Medical Affairs' strategies/tactics for SMA.
  • Develops and leads regional engagement plans with Key Opinion Leaders (KOLs) and other key external stakeholders that are aligned with the US Medical Affairs plan.
  • Maintains in-depth understanding of the disease state, product, competitors, marketplace, related medical areas, and regulatory guidelines.
  • Acts as the primary resource to healthcare professionals who request information on AveXis products, ensuring accurate and appropriate responses.
  • Partners and collaborates effectively with internal business partners.
  • Provides timely insights on emerging clinical / scientific trends back to the organization along with opportunities aimed at better meeting the needs of the external healthcare community.
  • Appropriately responds to unsolicited requests for clinical data per guidance from AveXis leadership.
  • Delivers timely, fair balanced, objective, scientific and economic information and education to health care customers, managed markets/healthcare/payer systems, and to internal business partners as required by business needs.
  • Partners with clinical operations to provide clinical sites with educational support.
  • Collaborates with Medical Information to assure medical information materials are up to date, accurate, fair-balanced, and clinically useful for stakeholders.
  • Supports the planning and execution of medical meetings (e.g., advisory boards, symposia, conferences, etc.).
  • Actively contributes to the execution of AveXis's strategy at scientific meetings by coordinationg RMD meeting/booth coverage and KOL meetings.
  • Leverages scientific knowledge and KOL relationships to identify opportunities for activities leading to scientific advancement.
  • Supports Investigator Initiated research studies through review, submission, evaluation, and recommendation of concepts / proposals.
  • Collaborates with internal Patient Advocacy relations function to serve as a primary field contact for national and regional patient and disease state associations and organizations.
  • Provides medical input into Health Economics and Outcomes Research (HEOR), disease models, epidemiological studies, market research and real-world study proposals.
  • Manages budget responsibilities efficiently regarding controllable expenses and performs all administrative requirements in a timely, accurate, and compliant manner.
  • Geography includes key SMA institutions in, but not limited to, the locations listed in the job title.


  • PharmD/PhD/MD or other professional terminal degree and commensurate experience.
  • Board Certified/Board Eligible genetic counselor preferred.
  • Minimum of three years of Medical Affairs experience (ideally as an MSL and preferably in the biotech industry) is required. Experience managing MSLs would be impactful.
  • Experience working in rare or orphan disease space.
  • Neurology, Neurodegenerative disease and/or pediatric specialty experience preferred.
  • Experience with Payer organizations preferred.
  • Excellent written and oral communication skills, including strong formal presentation skills.
  • Excellent planning and organization skills.
  • Strong interpersonal skills commensurate with the need to work closely with both external physicians/scientists and numerous internal business partners.
  • Ability to maintain the highest degree of integrity, represent the company's high ethics, moral behavior, and professionalism.
  • High proficiency in problem solving and strong scientific analytical skills.
  • Willingness/ability to travel up to 60 - 70% and internationally at times is required.

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.