Tanja Obradovic, Oncology Medical Strategy Advisor at Mercurial AI, shared a post on LinkedIn:
“Major Move: Biosimilars Policy in the US
FDA Opens Doors to Approval Without Clinical Efficacy Data, Big Possible Impact in Oncology
For the first time ever this month FDA accepted to waive clinical efficacy studies for the monoclonal antibody biosimilar. Illustrative example is biosimilar for Stelara (ustekinumab), an immunosuppressant used for conditions such as Crohn’s disease and psoriasis.
Instead of requiring clinical efficacy study(s), FDA agreed that totality of evidence based on comprehensive analytical similarity testing and immunogenicity, pharmacokinetics and pharmacodynamics studies will be sufficient to demonstrate biosimilarity (PR Newswire. Professor Sarfaraz K. Niazi Secures First-Ever FDA Acceptance to Waive Clinical Efficacy Studies for Monoclonal Antibody Biosimilars. 2025.)
Further strengthening new position, FDA issued few days ago updated Guidance ‘Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations Guidance for Industry’ that replaces guidance issued in 2019.
Major change is inclusion of statements that FDA may decide to waive clinical efficacy study for therapeutic proteins based on comprehensive package of data provided for analytical, toxicology, pharmacodynamics, immunogenicity and other data supportive of biosimilarity.
Some estimates put savings in biologic biosimilar development cost at 90% as clinical efficacy studies are expensive and can typically be at hundreds of millions of dollars while historically rarely uncovering meaningful differences from the reference product. Also, time to bedside will be significantly accelerated.
In totality, big benefits are likely for patients and health systems as this could lower drug costs, speed up approvals and broaden access to affordable biologics treatments worldwide. FDA position this month joins similar earlier developments in Europe and UK as the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) have already signaled support for reducing or eliminating CES requirements in certain biosimilar cases.
Impact can be huge and especially for very expensive Oncology immunotherapies that are starting to lose patents in 2028. Many biosimilars for PD-1 orignal inhibitors are in the pipeline with several in ongoing efficacy Phase III trials. Sponsors with already advanced analytical and toxicology packages may decide to accelerate strategy with urgent pharmacokinetics and immunogenicity part of the data summarization while newcomers can fully utilize policy changes from the start.
So the number of possible biosimlars for these expensive cancer immunotherapies coming onto market in 2028 may be expected to drastically increase resulting in higher pressure on pricing for originators and more competition among biosimilars.”
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