SVP Regulatory Affairs
uniQure
4 hours ago
Remote friendly (Lexington, MA)
United States
Corporate Functions
Key result areas (major duties, accountabilities and responsibilities)
Global Regulatory Strategy
- Develop, own, and execute the global regulatory strategy for AMT-130 and all pipeline programs, including filings with the FDA, MHRA, EMA, and other international authorities.
- With the CMO, lead all FDA/CBER interactions, including Type A, Type B, and pre-BLA meetings; serve as the company’s senior regulatory voice with health authorities.
- Architect parallel international regulatory pathways, with priority on UK MHRA and EU/EMA strategies.
- Weigh into regulatory and R&D pathways for earlier-stage pipeline assets (e.g., TLE, Fabry, future indications) by shaping IND/CTA strategies and target product profiles.
- Advise the CEO, CMO, Board, and executive team on regulatory risk, strategy, and health authority feedback; translate regulatory dynamics into executive decision options.
Label Strategy, Indication Expansion, and Cross-Organizational Partnership
- Partner with the SVP, Clinical Development on protocol design, endpoint selection, and evidence strategies to maximize regulatory success and label breadth.
- Build partnerships with Medical Affairs, Market Access, and Commercial to align regulatory strategy, labeling, and health authority engagement with positioning and patient access goals.
- Provide regulatory input across the full portfolio lifecycle to inform go/no-go decisions and portfolio prioritization.
- Serve as a core member of the CMO’s leadership team and key cross-functional partner to bridge research, development, and commercial functions.
BLA/MAA Filing and Submissions
- Lead preparation and submission of BLA, MAA, and other marketing applications; oversee regulatory components of eCTD/CTD in coordination with Clinical, CMC, Nonclinical, and Biostatistics.
- Manage regulatory aspects of external control methodology and novel statistical approaches with Biostatistics and external advisors.
- Ensure Phase III regulatory readiness, including protocol design input, endpoint strategy, and adaptive design considerations.
- Lead post-submission interactions with health authorities (information requests, advisory committee preparation, approval negotiations).
International Regulatory and Commercial Regulatory Infrastructure
- Build multi-jurisdiction filing strategy leveraging MHRA, EMA, and other approvals for market access (e.g., Saudi Arabia/SFDA, Gulf states) using formal reliance pathways.
- Partner to develop commercial regulatory infrastructure for launch, including labeling strategy, post-marketing commitments, and lifecycle management.
Regulatory Policy and Intelligence
- Monitor and advise on the global regulatory landscape for gene therapies/ATMPs, including FDA/CBER policy shifts and international harmonization.
- Engage with industry consortia, regulatory policy groups, and advocacy organizations to shape the external regulatory environment.
Qualifications & Skills
Essential Requirements
- Advanced degree (PhD, PharmD, MD, or equivalent) in a relevant scientific discipline.
- 15+ years progressive regulatory affairs experience in biopharmaceuticals; 10+ years in rare disease or gene therapy product development.
- Led at least one successful BLA or MAA through marketing authorization for a biologic or gene therapy product.
- Experience interacting with FDA/CBER, including leading Type A, Type B, and pre-BLA/pre-MAA meetings; global filing experience with MHRA, EMA, or other major agencies.
- Regulatory strategy experience across the full development lifecycle, including earlier-stage research/preclinical assets, IND/CTA-enabling programs, and late-stage registration.
- Strong leadership experience managing teams and elevating inherited teams; exceptional communication with experience presenting to regulators, boards, and executives.
Strongly Preferred
- Experience with external control and/or real-world evidence–based regulatory strategies.
- Experience with neurology or CNS-targeted therapies.
- Track record building partnerships across organizational boundaries (e.g., CMC, clinical, commercial, medical affairs, market access).
- Understanding of CMC requirements for gene therapy products (vector manufacturing, potency assays, comparability).
- Experience with regulatory aspects of surgical/procedure-based therapies (e.g., intracranial delivery, intrathecal administration).
- Prior experience in a publicly traded, rare disease–focused biotechnology company.
- Experience partnering with research and early development to shape regulatory strategy for preclinical and IND-stage programs across modalities/indications.
Personal Attributes
- Resilient and composed under pressure; comfortable with regulatory ambiguity and maintaining strategic clarity/team morale.
- Patient-centered.
- Strategically assertive; constructively advocates with regulatory agencies while maintaining relationships.
- Organizationally fluent; builds trust and alignment across commercial, medical affairs, and market access.
- Entrepreneurial; energized by pace and impact in a mid-cap biotech advancing first-in-class programs.
Salary Range
- $400,000 - $450,000
Global Regulatory Strategy
- Develop, own, and execute the global regulatory strategy for AMT-130 and all pipeline programs, including filings with the FDA, MHRA, EMA, and other international authorities.
- With the CMO, lead all FDA/CBER interactions, including Type A, Type B, and pre-BLA meetings; serve as the company’s senior regulatory voice with health authorities.
- Architect parallel international regulatory pathways, with priority on UK MHRA and EU/EMA strategies.
- Weigh into regulatory and R&D pathways for earlier-stage pipeline assets (e.g., TLE, Fabry, future indications) by shaping IND/CTA strategies and target product profiles.
- Advise the CEO, CMO, Board, and executive team on regulatory risk, strategy, and health authority feedback; translate regulatory dynamics into executive decision options.
Label Strategy, Indication Expansion, and Cross-Organizational Partnership
- Partner with the SVP, Clinical Development on protocol design, endpoint selection, and evidence strategies to maximize regulatory success and label breadth.
- Build partnerships with Medical Affairs, Market Access, and Commercial to align regulatory strategy, labeling, and health authority engagement with positioning and patient access goals.
- Provide regulatory input across the full portfolio lifecycle to inform go/no-go decisions and portfolio prioritization.
- Serve as a core member of the CMO’s leadership team and key cross-functional partner to bridge research, development, and commercial functions.
BLA/MAA Filing and Submissions
- Lead preparation and submission of BLA, MAA, and other marketing applications; oversee regulatory components of eCTD/CTD in coordination with Clinical, CMC, Nonclinical, and Biostatistics.
- Manage regulatory aspects of external control methodology and novel statistical approaches with Biostatistics and external advisors.
- Ensure Phase III regulatory readiness, including protocol design input, endpoint strategy, and adaptive design considerations.
- Lead post-submission interactions with health authorities (information requests, advisory committee preparation, approval negotiations).
International Regulatory and Commercial Regulatory Infrastructure
- Build multi-jurisdiction filing strategy leveraging MHRA, EMA, and other approvals for market access (e.g., Saudi Arabia/SFDA, Gulf states) using formal reliance pathways.
- Partner to develop commercial regulatory infrastructure for launch, including labeling strategy, post-marketing commitments, and lifecycle management.
Regulatory Policy and Intelligence
- Monitor and advise on the global regulatory landscape for gene therapies/ATMPs, including FDA/CBER policy shifts and international harmonization.
- Engage with industry consortia, regulatory policy groups, and advocacy organizations to shape the external regulatory environment.
Qualifications & Skills
Essential Requirements
- Advanced degree (PhD, PharmD, MD, or equivalent) in a relevant scientific discipline.
- 15+ years progressive regulatory affairs experience in biopharmaceuticals; 10+ years in rare disease or gene therapy product development.
- Led at least one successful BLA or MAA through marketing authorization for a biologic or gene therapy product.
- Experience interacting with FDA/CBER, including leading Type A, Type B, and pre-BLA/pre-MAA meetings; global filing experience with MHRA, EMA, or other major agencies.
- Regulatory strategy experience across the full development lifecycle, including earlier-stage research/preclinical assets, IND/CTA-enabling programs, and late-stage registration.
- Strong leadership experience managing teams and elevating inherited teams; exceptional communication with experience presenting to regulators, boards, and executives.
Strongly Preferred
- Experience with external control and/or real-world evidence–based regulatory strategies.
- Experience with neurology or CNS-targeted therapies.
- Track record building partnerships across organizational boundaries (e.g., CMC, clinical, commercial, medical affairs, market access).
- Understanding of CMC requirements for gene therapy products (vector manufacturing, potency assays, comparability).
- Experience with regulatory aspects of surgical/procedure-based therapies (e.g., intracranial delivery, intrathecal administration).
- Prior experience in a publicly traded, rare disease–focused biotechnology company.
- Experience partnering with research and early development to shape regulatory strategy for preclinical and IND-stage programs across modalities/indications.
Personal Attributes
- Resilient and composed under pressure; comfortable with regulatory ambiguity and maintaining strategic clarity/team morale.
- Patient-centered.
- Strategically assertive; constructively advocates with regulatory agencies while maintaining relationships.
- Organizationally fluent; builds trust and alignment across commercial, medical affairs, and market access.
- Entrepreneurial; energized by pace and impact in a mid-cap biotech advancing first-in-class programs.
Salary Range
- $400,000 - $450,000