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Global Medical Affairs Therapy Area Lead - PKU (East Coast Based Candidates)

PTC Therapeutics, Inc
United States
Jan 16, 2026

PTC Therapeutics is a global commercial biopharmaceutical company. For over 25 years our team has been deeply committed to a unified purpose: Extending life's moments for children and adults living with a rare disease.

At PTC, we cultivate an inclusive culture where everyone feels valued, respected, and empowered. We welcome candidates from all backgrounds to join our team, fostering a strong sense of belonging.

Visit our website to learn more about our company and culture!
Site: www.ptcbio.com

Job Description Summary: Global Medical Affairs (GMA) at PTC is committed to helping health care providers make evidence-based decisions to optimize health outcomes in their patients with rare diseases. The Global Medical Affairs - Global Therapeutic area Lead, PKU is responsible for defining the Global Medical Affairs vision, strategy and tactics to support PTC's PKU business.

He/she will partner with the Regional Medical Directors to identify and develop relationships with key opinion leaders (KOLs), identify treatment centers, ensure that the global medical affairs strategies and tactics for PKU are adopted and implemented at the local level and to ensure that the Global vision, strategy and tactics meet local needs.

He/she collaborates with marketing colleagues, to ensure that the strategic PKU medical affairs plan is aligned with the brand strategy.

He/she will partner with the global medical affairs (GMA) enabling functions (i.e. Publications, Medical Information, Medical Education, Medical Communications, etc.) to lead and execute the medical strategy for sepiapterin, which includes: updating/executing established medical plan, maintaining the scientific communication platform (publications, congresses etc.), planning and executing scientific advisory boards, review and approval of all grants (including IIS requests) and creating and delivering educational presentations both internally and externally. This individual will represent GMA on existing program team for sepiapterin, providing input into clinical development initiatives related to sepiapterin and will also support all life-cycle management initiatives. This role is viewed as the medical affairs subject matter expert on PKU and is expected to play a key role in educating KOLs, physicians, and internal colleagues.

This position ensures adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate. Job Description:

Responsibilities:

  • Develops and leads strategic global medical affairs activities related to PKU to accelerate commercialization efforts in launched markets and support successful commercialization in other markets to launch.
  • Drives and supports communication and collaboration amongst regional and local Medical Affairs functions, GMA enabling functions and other teams in Commercial, Market Access, etc. to ensure well developed and executed PKU strategic plans; drives internal cross-functional partnerships for all PKU medical initiatives.
  • Serves as the PKU subject matter expert in Global Medical Affairs. Partners with medical and commercial training to educate internal and external colleagues on the scientific and clinical aspects of PKU.
  • Develops and finalizes global evidence generation plan in close collaboration with regional and country medical heads, GMA enabling functions and ensures alignment with other key internal stakeholders.
  • Plans and leads the Investigator Initiated Studies (IIS) strategy for PKU.
  • Effectively engages and builds relationships with PKU KOLs, internal and external stakeholders and healthcare professionals for the successful implementation of medical strategy for PKU; identifies and engages in scientific exchange with medical experts in the PKU therapeutic area.
  • Serves as the strategic partner with respect to PKU to Clinical Development, Commercial Development and Life-Cycle Management (LCM) teams.
  • Represents Global Medical Affairs at international and regional scientific congresses.
  • Collaborates closely with the Marketing function to provide medical input for commercial activities related to therapy.
  • Plans and leads the publication strategy for PKU.
  • Supervises medical information process and service from the PKU indication perspective in close collaboration with other functions in medical affairs; ensures Medical Services offerings are appropriately utilized for PKU.

Qualifications:

  • Advanced Degree Required (PharmD, PhD or MD) with a minimum experience of 10 years progressively responsible experience in the pharmaceutical, biotechnology or related environment, preferably with a minimum of 7 years in medical affairs.
  • Prior experience as a Global or Regional Medical Lead (supporting the U.S.) within a rare disease; experience within metabolism, strongly preferred
  • Experience supporting key markets (including US)
  • Proven track record of successful leadership and progression within a medical affairs department working from both a strategic and tactical standpoint.
  • Demonstrated ability to accurately and effectively evaluate medical/scientific literature and develop effective medical and communication strategies.
  • Ability to analyze data and synthesize a compelling scientific narrative tailored to relevant audiences.
  • Comprehensive understanding of compliance focus concerning medical affairs functions.
  • Demonstrated track record of success supporting the commercialization of an orphan, rare disease and/or specialized drug(s).
  • In-depth experience dealing with payer organizations.
  • Demonstrated ability to create and lead cross-functional teams.
  • Demonstrated ability to develop relationships with and influence KOLs, important customers, challenge common perceptions and implement medical affairs tools to improve patient care and outcomes.
  • Experience building and maintaining KOL network and interacting with patient organizations.
  • Ability to influence without direct authority.
  • Proficiency with Microsoft Office.
  • Excellent verbal and written communication and skills.
  • Ability to take complex scientific information and communicate it clearly and effectively to diverse audiences in a clear and concise manner
  • Ability to work effectively in a global, multicultural environment
  • Ability to work independently and collaboratively, as required, in a fast-paced, matrixed, team environment consisting of internal and external team members.
  • Analytical thinker with excellent problem-solving skills and the ability to adapt to changing priorities and deadlines.
  • Excellent planning, organization and time management skills including the ability to support and prioritize multiple projects.
  • Fluent in English (verbal and written).

*Special knowledge or skills and/or licenses or certificates preferred.

  • Expertise on PKU or related inborn errors of metabolism
  • Experience in orphan/rare diseases, neurology and/or genetic disorders.
  • Knowledge of US Market (payors and KOL interactions)

Travel requirements:

40% global travel

Office-based or remote position with travel to PTC offices and off-site meetings as needed. Candidates must be based in the U.S. (and preferably East-coast).

Expected Base Salary Range:

$265,700 - $334,500. The base salary offered will be contingent on assessment of candidate education level, background, and experience relative to the requirements of the position they are being considered for, as well as review of internal equity.

In addition to base salary, PTC employees are also eligible for short- and long-term incentives.All eligible employees may also enroll in PTC's medical, dental, vision, and retirement savings plans

EEO Statement:

PTC Therapeutics is an equal opportunity employer. We welcome applications from all individuals, regardless of race, color, national origin, gender, age, physical characteristics, social origin, disability, religion, family status, pregnancy, sexual orientation, gender identity, gender expression, disability, veteran status or any unlawful criterion under applicable law. We are committed to treating all applicants fairly and avoiding discrimination.

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