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Zevra Therapeutics

Develops rare-disease therapies, including CNS

Senior Medical Director - U.S. Medical Affairs

Biology & BiotechUpdated on Nov 14
Full-timeBoston, MA, USANo salary listed

About the Job

Requirements

  • An MD, PhD, or equivalent degree is required.
  • At least 7 years of relevant Medical Affairs experience in the biotechnology or pharmaceutical industry, with progressive responsibility and demonstrated medical leadership.
  • Deep scientific and medical expertise in rare diseases, including understanding of rare disease treatment landscapes, clinical development, and unmet medical needs.
  • Significant experience leading the development and execution of U.S. Medical Affairs strategy, preferably for commercial-stage products, with demonstrated ownership of medical plans and initiatives.
  • Strong experience in scientific exchange, key opinion leader engagement, medical communications, and evidence generation.
  • Ability to critically evaluate and interpret complex clinical data and translate scientific insights into actionable medical strategies.
  • Experience collaborating cross-functionally with Commercial, Market Access, Patient Advocacy, and other internal stakeholders.
  • Demonstrated leadership capabilities and interest in managing and developing a field-based Medical Science Liaison team alongside broader corporate Medical Affairs responsibilities.
  • Ability to operate effectively in a dynamic, growing biotechnology environment.

Responsibilities

  • Develop, lead, and execute the U.S. Medical Affairs strategy for MIPLYFFA, aligning it with organizational priorities, evolving scientific evidence, and the needs of patients and healthcare professionals.
  • Serve as a senior medical and scientific expert in Niemann-Pick disease type C, maintaining a deep understanding of the disease landscape, clinical data, treatment considerations, and emerging scientific developments.
  • Provide medical leadership and scientific guidance across U.S. Medical Affairs initiatives, including medical education, scientific communications, evidence generation, and external engagement.
  • Establish and maintain relationships with key opinion leaders, healthcare professionals, investigators, and other external scientific stakeholders to facilitate scientific exchange and identify unmet medical needs.
  • Lead and support evidence generation initiatives, including Phase IV studies, registries, investigator-initiated studies, real-world evidence initiatives, and expanded access programs, as appropriate.
  • Partner cross-functionally with Commercial, Market Access, Patient Advocacy, and other internal stakeholders to provide medical expertise and support compliant, scientifically sound business initiatives.
  • Participate in Medical Review Committee and Promotional Review Committee processes, providing medical and scientific review of relevant materials to ensure accuracy, scientific integrity, and compliance.
  • Review scientific publications, abstracts, presentations, medical education materials, promotional and non-promotional content, reimbursement dossiers, and payer-related materials.
  • Support development and execution of medical communications and educational initiatives to advance understanding of Niemann-Pick disease type C, MIPLYFFA, and the broader rare disease landscape.
  • Provide medical and scientific input into payer engagement activities, including communication of clinical evidence and development of scientifically accurate resources supporting patient access.
  • Translate scientific developments, external stakeholder insights, and emerging clinical evidence into actionable recommendations that inform U.S. Medical Affairs priorities.
  • Recruit, build, and lead a high-performing U.S. field-based Medical Science Liaison team, providing ongoing coaching, mentoring, performance management, and professional development to ensure scientific excellence and alignment with Medical Affairs objectives.
  • Ensure field medical activities and scientific engagement strategies align with U.S. Medical Affairs objectives and are executed with scientific rigor and compliance.
  • Partner with Medical Excellence to establish and evaluate appropriate metrics, field medical insights, and performance indicators to support continuous improvement.
  • Provide medical expertise and scientific training to internal teams, including field medical and cross-functional stakeholders, as appropriate.
  • Maintain scientific integrity, ethical conduct, and compliance with applicable regulatory requirements, industry standards, and company policies.

Desired Qualifications

  • Prior people management experience.

About the Company

Zevra Therapeutics

Develops rare-disease therapies, including CNS

Zevra Therapeutics develops and commercializes therapies for rare diseases, especially rare CNS disorders, by acquiring assets and pursuing in-house development. Its products include OLPRUVA for urea cycle disorders and azstarys, a once-daily ADHD treatment marketed in the U.S. with Corium for commercialization, plus a development pipeline like arimoclomol for Niemann-Pick disease type C. The company mixes internal R&D with strategic acquisitions and engages with regulators, patients, and advocacy groups to target underserved populations. Its goal is to build a differentiated portfolio of therapies that deliver sustainable value for patients and stakeholders.

Company size
51-200
Company stage
IPO
Headquarters
Boston, MA
Founded
2006

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