FDA Grants First Ever Waiver of Clinical Efficacy Studies for Monoclonal Antibody Biosimilar

• The FDA has granted the first-ever waiver of clinical efficacy studies for monoclonal antibody biosimilars, signaling a fundamental shift toward analytics-driven approval pathways.
• The FDA’s decision is expected to reduce the cost of developing biosimilars by more than 90%, removing one of the biggest barriers to market entry. Approval times could be reduced by more than 70%, providing patients around the world with faster access to affordable biologic medicines.
• This exemption lowers costs, shortens timelines, and allows biotech firms to pursue more ambitious strategies, from portfolio diversification to new market expansion. Smaller biotech firms, once constrained by the high costs of CES, now have greater opportunities to enter the biosimilar market and compete with established players. CDMOs will become indispensable partners, offering the infrastructure and regulatory expertise required to seize this market opportunity effectively.
• By relying on advanced analytical and PK/PD data instead of redundant clinical trials, regulators are accelerating the pace of biosimilar innovation and patient access. FDA decision removes one of the most costly and time-consuming barriers in biosimilar development, making entry into the market more financially viable for both small and large biotechnology companies.

In a move set to redefine biosimilar drug development, the FDA has waived the need for clinical efficacy studies (CES) in the case of monoclonal antibody biosimilars. This first-ever decision is the culmination of years of scientific advocacy, highlighting that the era of mandatory CES may be coming to an end. Instead, regulators are placing their trust in the rigor of analytical and PK/PD data.

This development is highly significant for the biopharmaceutical sector. For years, CES requirements posed one of the greatest challenges in biosimilar development. They demanded extensive patient trials, which not only prolonged timelines but also duplicated existing clinical knowledge from the reference product. By removing this requirement in suitable cases, the FDA is signaling a shift toward smarter, science-driven regulation.

For biotechnology companies, the implications extend well beyond cost savings. The CES exemption unlocks new strategies for market participation, particularly for smaller or mid-sized firms. Instead of allocating disproportionate resources to clinical efficacy studies, these companies can channel investment into innovation, expanding product pipelines, and pursuing geographic expansion. The removal of CES as a mandatory requirement makes biosimilar development more financially viable, stimulating greater market entry and intensifying competition. Established companies, too, stand to benefit by reallocating capital toward differentiation strategies and global launches. In both cases, the FDA’s decision empowers biotech firms to pursue more ambitious goals and adapt faster to evolving patient needs.

The implications for CDMOs are equally transformative. As development pathways become less clinical-trial heavy and more reliant on advanced analytics and manufacturing precision, biotech companies will increasingly lean on CDMOs for expertise. CDMOs capable of offering integrated services will find themselves at the heart of strategic partnerships. For smaller biotech firms lacking infrastructure, CDMOs act as enablers, providing the technical capacity and regulatory guidance needed to succeed in competitive markets. For larger companies, CDMOs bring scalability and flexibility, allowing them to accelerate timelines without compromising quality. In this new paradigm, collaboration between biotech innovators and CDMOs is not optional but a critical driver of market competitiveness and patient impact.

By reducing barriers and fostering collaboration, the FDA’s decision is a win-win: patients benefit from faster access to affordable biologics, while biotech firms and CDMOs unlock new growth opportunities. This milestone sets a precedent for broader global adoption of similar policies, accelerating the future of biosimilars.

Prepared by:

Jakub Knurek

Marketing Specialist

j.knurek@mabion.eu

Jakub Knurek

Marketing Specialist

Jakub Knurek is a marketing and business development expert specializing in CDMO operations with scientific and biopharmaceutical background. During the COVID-19 pandemic, he contributed to the development of the Novavax protein vaccine. As a side project, he runs his own pharma company.

As the author of numerous articles, he is recognized as an important voice of the younger generation of scientists. He is a member of Mabion’s representation at industry trade shows, including BIO International Convention 2025 in Boston, 2nd Global Biologics India 2025 in Hydrabad, CPHI 2024 in Milan, CPHI 2023 in Barcelona, Life Sciences Baltics 2023 in Vilnius. He actively participates in academic, business and industry discussions, such as the “Building the Future Pharma Workforce” panel at CPHI Milan 2024.

Sources and further reading

1. PR Newswire. Professor Sarfaraz K. Niazi Secures First-Ever FDA Acceptance to Waive Clinical Efficacy Studies for Monoclonal Antibody Biosimilars. 2025.
2. Niazi S. Scientific Rationale for Waiving Clinical Efficacy Testing of Biosimilars. Drug Des Devel Ther. 2022; 16: 2803-2815.
3. Niazi S. Support for Removing Pharmacodynamic and Clinical Efficacy Testing of Biosimilars: A Critical Analysis. Clin Pharmacol Drug Dev. 2023; 12(12): 1134-1141.