PTC’s 2025 Corporate Social Responsibility (CSR) Report captures a year defined by focus, execution and measurable progress toward our mission: delivering clinically differentiated medicines for children and adults living with rare disorders.
Read the full 2025 CSR Report to explore our progress and ongoing commitments.
Advancing patient treatments through innovation and access
A major milestone in 2025 was the U.S., EU, and Japan regulatory approvals and strong initial global launch of SephienceTM, our oral therapy for children and adults living with PKU. Broad early uptake across age groups and disease severities underscores both the significant unmet need in PKU and PTC’s ability to execute with speed, scale, and reach.
PTC’s global infrastructure continues to support access to therapies in 50+ countries, using a combination of direct operations, partnerships, and distributors. The report emphasizes that access is not a one-time event, but a continuum supported by market-specific planning, engagement with regulators and payers, and patient support programs designed to help patients initiate and remain on therapy within complex healthcare systems.
Strengthening our pipeline and scientific platforms
The CSR Report also spotlights continued investment in R&D through our two innovative scientific platforms: Splicing and Inflammation & Ferroptosis, with programs in areas such as Huntington’s disease, myotonic dystrophy, neurodegenerative diseases, Parkinson’s disease, inflammatory lung disease and autoimmune conditions.
Putting people at the center: patients, employees and communities
PTC’s people and culture enable execution in 2025. The report highlighted our patient focus, spotlighting our collaboration with 200+ patient advocacy organizations worldwide and dedicated regional advocacy teams supporting patients and families throughout the rare disease journey. In the U.S., PTC Cares™ continued to provide case management support to help eligible patients access treatment and navigate complex healthcare systems.
Our global workforce continued to power our performance as the company continued investing in talent attraction, learning and development. The report shows that employee engagement remained strong, with employees rating their likelihood to recommend PTC 4.73 out of 5, and voluntary turnover staying below 4% over the last three years. Nearly 43% of employees participated in at least one CommUNITY Resource Group in 2025, reinforcing our inclusion-focused culture.
Community impact and environmental responsibility
PTC supported rare-disease and science-related communities through 100+ grants and charitable donations, contributing nearly $4.7 million globally to patient advocacy, continuing medical education, and independent research support, plus approximately $60,000 invested in STEM education programs near our New Jersey and California offices.
Operating responsibly: environment, governance and integrity
The report outlines an evolving, risk-informed environmental approach and highlights practical actions taken in 2025, such as renewable electricity sourcing, waste reduction, circular-economy practices during site moves and 1,132 trees planted through “Planting the PTC Forest.”
Strong governance and risk management remain embedded across the organization through Board oversight and ethical business conduct expectations, reinforcing transparency and accountability across our operations.