2025 年 9 月 1 日，在全球医疗健康与制药创新领域，一项具有里程碑意义的突破诞生：伊利诺伊大学芝加哥分校兼职教授、多家生物制药企业创始人萨尔法拉兹・K・尼亚齐（Sarfaraz K. Niazi）教授首次获得美国食品药品监督管理局（FDA）认可，其研发的单克隆抗体生物类似药可免做临床疗效研究（CES）。这一决定从根本上重新定义了生物药的研发、审批模式，并将推动全球患者更易获得价格可负担的生物药。

数十年来，尽管临床疗效研究都证明生物类似药有效，但它一直是生物类似药审批流程中的核心环节，且成本高昂。尼亚齐教授曾多次向 FDA 提交公民请愿书，并发表数十篇经同行评审的论文为 FDA 提供建议。他在这些材料中始终主张，临床疗效研究对生物类似药的评估并无科学价值，因为分析相似性研究和免疫原性研究已足以证明单抗的生物类似性。如今，他的倡导终见成效，FDA 认可了这一基于证据的范式转变。

首个案例：喜达诺（Stelara）生物类似药

尼亚齐教授针对喜达诺（Stelara，通用名：乌司奴单抗，ustekinumab）的生物类似药申请，将成为首个无需任何临床测试即可向 FDA 提交的申请，这标志着监管领域迎来突破性先例。

对全球药品可及性的影响

取消临床疗效研究要求，预计将带来以下改变：

• 使生物类似药的研发成本降低 90% 以上

• 使审批周期缩短 70% 以上

• 助力中小型企业进入生物类似药市场（此前该市场准入主要局限于资源雄厚的大型制药企业）

• 推动定价竞争，使其价格走势向小分子仿制药靠拢，让更多患者能负担得起此类药物

这一决定也使 FDA 与欧洲药品管理局（EMA）及英国药品和医疗产品监管局（MHRA）的政策保持一致 ，这两个机构此前已根据尼亚齐教授的建议采取了相关措施。随着各监管辖区政策的协调统一，全球生物类似药的审批路径比以往任何时候都更加清晰。

“开创先河” 的职业生涯

尼亚齐教授在推动行业变革方面早已成绩斐然，其贡献包括：

• 首创 “生物类似药”（biosimilars）这一术语，创立美国首家生物类似药公司，并推动多款生物类似药获得 FDA 批准

• 通过改善药物穿越血脑屏障的能力，研发出首个有效的阿尔茨海默病治疗方案

• 在《科学》杂志发表论文后，推动美国国会批准生物药免做动物实验

• 对 2024 年诺贝尔化学奖评选委员会提出质疑，并指出《科学》杂志上一篇关于人工智能主题的论文存在错误

历史性转折点

尼亚齐教授表示：“这不仅是我的胜利，更是科学的胜利、理性的胜利，最重要的是，是患者的胜利 ，他们理应获得价格可负担的生物药。FDA 的认可开启了一个新时代，在这个时代，创新不再受过时测试要求的束缚。这将从根本上重塑生物类似药的经济格局，并扩大其在全球的可及性。”

随着全球各监管机构持续协调政策，FDA 今日的这一决定，标志着现代药物研发领域迎来了最具历史意义的转折点之：严谨的科学、患者可及性与成本降低在此实现了交汇。

目前，尼亚齐教授正鼓励中小型仿制药企业进入生物类似药领域，因为该领域的研发成本现已降至这些企业可轻松承担的水平。这将大幅降低生物类似药的价格（这也是研发生物类似药的首要目标），让全球大多数患者都能负担得起。

Professor Sarfaraz K. Niazi Secures First-Ever FDA Acceptance to Waive Clinical Efficacy Studies for Monoclonal Antibody Biosimilars

Sept. 1, 2025-- In a groundbreaking milestone for global healthcare and pharmaceutical innovation, Professor Sarfaraz K. Niazi, Adjunct Professor at the University of Illinois at Chicago and founder of multiple biopharmaceutical enterprises, has secured the first-ever FDA acceptance to waive clinical efficacy studies (CESs) for monoclonal antibody biosimilars. This decision fundamentally redefines how biological drugs will be developed, approved, and made affordable for patients worldwide.

For decades, CESs have been a central — and costly — component of biosimilar approval, despite their inability ever to demonstrate meaningful failure outcomes. In numerous citizen petitions to the FDA and dozens of peer-reviewed publications advising the FDA, Professor Niazi has persistently argued that CESs add no scientific value to biosimilar evaluation, as analytical similarity and immunogenicity studies are sufficient to confirm biosimilarity. His advocacy has now culminated in the FDA's acceptance of this evidence-based paradigm shift.

The First Case: Stelara Biosimilar

Professor Niazi's biosimilar application for Stelara (ustekinumab) will be the first filed with the FDA without requiring any clinical testing, marking a precedent-setting regulatory breakthrough.

Global Impact on Drug Affordability

The removal of CES requirements is expected to:

Reduce biosimilar development costs by over 90%

Accelerate approval timelines by more than 70%

Empower small- and mid-sized companies to enter the biosimilar market, where entry had previously been limited to resource-rich pharmaceutical giants

Spur competitive pricing that mirrors the trajectory of generic small-molecule drugs, delivering widespread affordability to patients

This decision aligns the FDA with the European Medicines Agency (EMA) and the UK's MHRA, which have already taken steps in line with Niazi's recommendations. With harmonization across regulatory jurisdictions, the pathway to global biosimilar approvals becomes clearer than ever before.

A Career of Firsts

Professor Niazi is no stranger to pioneering change. His contributions include:

Creating the term "biosimilars," establishing the first US biosimilar company, and securing FDA approval of multiple biosimilars.

Creating the first effective treatment of Alzheimer's disease by improving its entry across the blood-brain barrier.

Securing the US Congress approval of a waiver of animal testing of biological drugs after publishing his thesis in Science Magazine.

Challenging the Nobel Prize Committee for the 2024 Chemistry Prize, and pointing out the mistake in publishing a wrong paper in Science Magazine on the topic of artificial intelligence.

A Historic Turning Point

"This is not just my victory," said Professor Niazi. "This is a victory for science, for reason, and most importantly, for patients who deserve affordable access to biological medicines. The FDA's acceptance ushers in a new era where innovation is not held hostage by outdated testing requirements. It will fundamentally reshape the economics of biosimilars and expand access globally."

As regulators worldwide continue to align, today's decision by the FDA marks one of the most historic turning points in modern drug development — where rigorous science, patient access, and cost reduction converge.

Professor Niazi is now encouraging mid-sized and small generic companies to enter the field of biosimilars, as the cost of their development will be readily affordable. This will allow for substantial price reduction, the primary goal of biosimilars, and make them affordable to the majority of global patients.