FDA accepts first biosimilar without clinical efficacy studies: Will this redefine global biologics access?

In a move set to alter the economics and regulatory landscape of biosimilars forever, the United States Food and Drug Administration (FDA) has, for the first time in its history, accepted a biosimilar application for a monoclonal antibody without requiring clinical efficacy studies (CESs). The application, submitted by Professor Sarfaraz K. Niazi—a veteran regulatory scientist, entrepreneur, and Adjunct Professor at the University of Illinois at Chicago—targets a biosimilar version of Stelara (ustekinumab), a blockbuster drug used in treating inflammatory diseases such as psoriasis, Crohn’s disease, and ulcerative colitis.

This decision is already being described by healthcare stakeholders as a “regulatory inflection point” and a “game-changer for biosimilar access,” potentially opening the floodgates for lower-cost alternatives to high-priced biological drugs. It represents not just a single-company milestone but a significant paradigm shift in how biosimilars are developed, approved, and delivered to patients.

The FDA’s decision effectively accepts the scientific framework Niazi has long argued—that comparative clinical efficacy studies offer little incremental value once robust analytical and functional comparability between the biosimilar and reference product has been demonstrated.

What does the FDA’s CES waiver for Stelara’s biosimilar mean for drug development costs and access?

Clinical efficacy studies have historically been one of the most expensive and time-consuming aspects of biosimilar development. For monoclonal antibodies, which are large and complex proteins, these trials could easily push development costs well above $150 million and extend timelines to 8–10 years. By eliminating CESs from the regulatory requirement in this landmark case, the FDA has cleared a path to cut biosimilar development costs by more than 90% and shave over 70% off the approval timeline, according to Niazi’s estimates.

This is more than a procedural update—it’s an economic revolution. The biosimilar industry has struggled for years to emulate the price-disruptive impact of small-molecule generics. With CESs no longer mandated, small and mid-sized biopharmaceutical firms now have a realistic opportunity to enter the monoclonal antibody segment, a market historically controlled by large incumbents with the capital to absorb long and expensive development cycles.

Professor Niazi emphasized that this regulatory evolution creates a fairer market landscape. With the FDA now aligning more closely with regulators like the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), the global regulatory ecosystem appears poised for harmonization. That, in turn, is expected to accelerate biosimilar launches across regions and promote true affordability for patients globally.

How are institutional stakeholders reacting to the FDA’s CES policy shift for biosimilars?

While the FDA has not issued a blanket removal of CES requirements, industry observers and institutional investors are treating the Stelara biosimilar precedent as a harbinger of policy liberalization. Analysts have suggested that sponsors seeking approval for biosimilars referencing well-characterized reference products may now consider submitting applications without clinical trials, especially when robust analytical similarity and immunogenicity data are available.

This development could also reshape the strategic outlook for generic giants like Viatris, Amgen, Samsung Bioepis, and Coherus BioSciences. These firms may now reevaluate internal R&D allocation, focusing instead on accelerated development of monoclonal antibody biosimilars without trial-based efficacy endpoints.

For venture-backed biosimilar startups and emerging-market players, the financial modeling becomes more attractive. Lower barriers to entry mean shorter capital cycles, faster go-to-market execution, and the ability to target niche or underserved therapeutic areas where biosimilar competition has been thin due to CES costs.

Portfolio managers and healthcare-focused institutional funds may begin revising exposure to biosimilar portfolios, particularly those positioned to pivot rapidly into CES-free development strategies. Regulatory consultancies and CROs may also adapt to accommodate this shift in dossier preparation, focusing more on analytical characterization and immunogenicity profiling services.

How does Professor Sarfaraz K. Niazi’s advocacy history shape the regulatory science behind this breakthrough?

Professor Sarfaraz K. Niazi has long occupied a unique position at the intersection of science, regulation, and policy. His biosimilar advocacy began decades ago, when he founded the first U.S.-based biosimilar company. He is widely credited with coining the term “biosimilars” and has authored multiple books and peer-reviewed articles on the scientific, regulatory, and economic aspects of biosimilar development.

Over the years, he has submitted several citizen petitions to the FDA, many of which directly addressed the scientific inefficiency and ethical concerns of CESs. His argument has been consistent: once a biosimilar has demonstrated sufficient analytical and immunogenicity similarity to a reference product, CESs become redundant. He also contended that these studies never produce failure outcomes due to their underpowered design, making them scientifically meaningless but financially burdensome.

Niazi’s thought leadership extends beyond biosimilars. His work on blood-brain barrier technologies has been recognized for advancing treatments in neurodegenerative disorders like Alzheimer’s. He also authored a pivotal thesis published in Science that catalyzed U.S. Congressional support for waiving animal testing for biologics—a victory that mirrors the ethos behind CES waivers.

Notably, he has challenged mainstream narratives in regulatory science, including questioning the Nobel Prize awarded for AI-based protein prediction tools like AlphaFold and Rosetta, citing fundamental flaws in the published methodologies. These efforts underscore his profile as both a scientist and a systems reformer.

Why is the FDA’s CES waiver being hailed as a turning point for global biosimilar access?

Biosimilars were created with the goal of offering affordable versions of biologic therapies once patents expired. However, that promise has largely gone unfulfilled in the U.S. due to the cost and complexity of development and regulatory approval. By requiring CESs, regulators unintentionally favored well-capitalized multinational drugmakers, restricting competition and limiting the pricing benefits that generics brought to the small-molecule space.

Today’s shift is being seen as a corrective measure that realigns regulatory frameworks with the original mission of biosimilars: to enhance access. The impact is potentially massive. As more biosimilars get approved under this new precedent, prices could fall dramatically—mirroring the steep cost drops seen in the generics market over the past three decades.

This could be especially significant for high-cost biologics used in chronic autoimmune diseases and oncology, where treatment costs often exceed tens of thousands of dollars per year per patient. If CES waivers become the norm, access to life-changing therapies could expand across developing nations, low-income patient groups, and even in publicly funded health systems seeking budget optimization.

Professor Niazi made clear that this milestone was not just a personal or academic victory, but a win for millions of patients globally. He has urged smaller pharmaceutical companies and generics developers to act quickly and enter the biosimilar space, leveraging the new regulatory simplicity to bring affordable monoclonal antibodies to market.

What could come next for biosimilar regulation following this Stelara precedent?

Experts believe the Stelara case will trigger a wave of similar submissions. Sponsors may soon test this approach with biosimilars referencing other top-selling biologics such as Humira (adalimumab), Keytruda (pembrolizumab), and Avastin (bevacizumab)—each with multi-billion dollar annual sales and vast therapeutic footprints.

Industry watchers are also anticipating increased lobbying to formalize the CES waiver into FDA guidance. If institutional data from this Stelara case and future applications show positive post-market outcomes, regulators may codify this approach for certain biologic classes.

Furthermore, the waiver may also open doors for technological innovations in biosimilar development, including AI-driven structural similarity assessments and next-generation immunogenicity prediction platforms.

For now, Professor Niazi and his regulatory consultancy, Pharmaceutical Scientist, Inc., are actively advising both regulators and manufacturers on how to navigate this new frontier. Their guidance is expected to shape not just U.S. submissions but global filings in jurisdictions influenced by FDA trends.