A Rare Disease CEO Responds to the Resignation of the FDA’s Peter Marks

Rare Daily Staff

Peter Marks, who has served as the director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research since 2016, resigned from his post over U.S. Health and Human Services Secretary Robert F. Kennedy, Jr. assault on the safety and efficacy of vaccines.

“Over the past 13 years I have done my best to ensure that we efficiently and effectively applied the best available science to benefit public health. As you are aware, I was willing to work to address the Secretary’s concerns regarding vaccine safety and transparency by hearing from the public and implementing a variety of different public meetings and engagements with the National Academy of Sciences, Engineering, and Medicine,” he wrote in his letter of resignation. However, it has become clear that truth and transparency are not desired by the Secretary, but rather he wishes subservient confirmation of his misinformation and lies.”

Though the controversy is focused on vaccines, Marks has been a critical figure in advancing new therapeutic approaches to treat rare, genetic diseases. He has also demonstrated a willingness to apply regulatory flexibility to balance the need to demonstrate safety and efficacy of experimental therapies with the seriousness of an unmet medical need. And, though his work with the National Institutes of Health on the Bespoke Gene Therapy Consortium, he has worked accelerate the time and lower the cost of developing N-of-1 therapies for deadly conditions without therapeutic options.

The rare disease community was shaken by the news of Marks’ resignation and views it as the loss of a significant ally from an organization that doesn’t always appreciate the unique circumstances posed by developing therapies for rare diseases.

Jeremy Levin, chairman and CEO of Ovid Therapeutics, posted a response to the news on LinkedIn. It is reprinted here with permission.

By Jeremy Levin

It is with grave concern that I share the attached resignation letter from Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research.

Peter Marks is a person of extraordinary integrity and moral courage—a leader whose career has exemplified the highest ideals of science, medicine, and public service. He has been deeply admired as a vigilant steward of the scientific rigor and ethical standards that underpin the FDA’s credibility. He has served the United States with distinction, and his leadership has benefited patients not only in America but across the world. He has helped build America into the world leader in Regulatory Sciences.

His resignation is far more than the departure of a highly respected figure. It is a stark and unequivocal warning. In this letter—especially in the final paragraphs—Dr. Marks makes clear that the FDA, long held as the gold standard in global regulatory science, is now under threat from political interference and the systematic distortion of scientific truth. His words are as courageous as they are devastating: truth and transparency are no longer welcome. What is demanded instead is obedience.

This letter should shake us all. It signals that the very infrastructure we rely on to evaluate, approve, and monitor life-saving medicines—and to protect patients from harm—is at risk of collapse. The implications are vast. The consequences for biomedical innovation, public trust, and America’s role as a global health leader are profound.

Many have warned that key institutions in the United States are facing unprecedented pressure. With Dr. Marks’ resignation, we now have confirmation that the FDA—an institution vital not only to American public health but to the health of billions worldwide—is being compromised. This threatens not only the safety and well-being of millions of Americans, but also the global reliance on U.S. leadership in setting standards, developing breakthrough therapies, and advancing the frontiers of modern medicine.

Please read Dr. Marks’ letter with the seriousness it demands. Share it widely—with colleagues, institutions, policymakers, and anyone who believes that truth, science, and public health must never be politicized. This is not merely a personal act of conscience—it is a clarion call to all who recognize the moral responsibility to safeguard human health, both in the United States and across the globe. Partisan politics has no place in the effort to conquer disease, protect patients, and ensure that medicines remain safe, effective, and guided by evidence. If we care about the future of medicine, we must speak up, stand together, and act to defend the institutions that make progress possible.