RFK Jr Sacking Peter Marks From FDA Has Changed the Game for Gene Therapies—It's Over!

About Peter Marks

Here is just one example of what Peter Marks was doing when Director at FDAs Center for Biologics Evaluation & Research (CBER):

Top FDA official Peter Marks overruled staff, review team to approve Sarepta gene therapy

“For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy.

On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year.”

In effect, anyone with the condition can get the gene therapy, even though it wasn’t proven to work. Does that sound familiar?

Yes, on Peter Marks’ watch, all at COVID gene therapies were waved through in a similar way.

Dr Martin Makary is the New FDA Commissioner

This is Dr Martin (Marty) Makary

“In this role, Dr. Makary oversees the full breadth of the FDA portfolio and execution of the Federal Food, Drug, and Cosmetic Act and other applicable laws. This includes assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices; the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation; and the regulation of tobacco products.”

Dr Makary is on a steep learning curve, as he has a career as a cancer surgeon, not regulation. However, the signs are very promising.

This is something I have just written in my PharmaFlow Newsletter:

FDA Brings Back Previously Disbanded Generic Drug Policy Panel

June 10, 2025 | Tristan Manalac

From the article above, published in BioSpace, we hear that:

"The reinstatement of the generic drug policy office is the latest reversal of course for Robert F. Kennedy Jr.'s HHS, which also recently rehired FDA staff responsible for making travel arrangements and those involved in user fee program negotiations."

The FDA has brought back its generic drug policy office after previously gutting the team, according to a Monday report from Endpoints News, marking the latest development in the Trump administration’s back-and-forth staffing measures."

What should be made of that?

Firstly, the pain and suffering of unexpected job loss should be recognized, especially if it was through no fault of the job holder. On LinkedIn these days, it is not unusual to see 'Open to Work' around the faces of former FDA employees. Adjusting to these changed circumstances is never easy, even if the eventual outcome is transition to a more satisfying career. As some also move into the broader industry, which appears to be the case, there will undoubtedly be societal benefits from a two-way exchange of skills and knowledge.

Returning to the article, there is a counter argument relating to the assertion of "the Trump administration’s back-and-forth staffing measures."

Major organizational change programs are fraught with difficulties and unanticipated outcomes. Whether you believe the reasons behind change are valid, or not, the emphasis must be on responding to actual observed outcomes, adjusting plans and resources to stay on track.

In my professional opinion, the adjustments we read of are legitimate responses to maintain focus on the FDAs mission-critical objectives.

Establishment inspections are crucial to the quality and integrity of the pharmaceutical supply chain. Not reinstating supporting resources would jeopardize patient safety. Along with that, existing funding of FDA activities would need to be maintained, as a minimum, requiring staffing to be returned to duty.

As regards this latest adjustment reinstating OGDP staff, there is clear justification for this. After 40+ years of the Hatch-Waxman Act, generic (and some biosimilar) drugs now account for 90% of the medicines patients get in the United States.

Yes, that's 90% of medicines consumed across the United States—no small amount by any measure!

Below are the responsibilities of OGDP taken from the FDA website link for the Office of Generic Drugs:

"Office of Generic Drug Policy (OGDP)

Kristin Davis, J.D., Director, Office of Generic Drug Policy

Christopher Pruitt, J.D., Deputy Director, Office of Generic Drug Policy

OGDP consists of its own Immediate Office and three Divisions:

DIVISION OF LEGAL AND REGULATORY SUPPORT (DLRS) – Advises OGD regarding generic drug application-specific legal, regulatory, and policy issues and serves as agency experts on Hatch-Waxman and other generic drug regulatory issues. Tracks and helps expedite review of public health priority submissions, including for first generics and drug shortages, to help achieve timely approval of generic drug products. Advises and makes decisions on patent and exclusivity matters for brand and generic drugs and determines timing of generic drug approval.

DIVISION OF ORANGE BOOK PUBLICATION AND REGULATORY ASSESSMENT (DOBPRA) - Publishes and maintains the publication “Approved Drug Products with Therapeutic Equivalence Ratings” (the Orange Book), which provides a range of information on brand and generic drugs, including information regarding market availability, substitutability, drug patents and exclusivity, and ensures timely and accurate information is included in the Orange Book regarding all new drug approvals and therapeutic equivalence evaluations for multi-source products.

DIVISION OF POLICY DEVELOPMENT (DPD) – Provides strategic leadership and direction in the development, clearance, and implementation of regulations, guidances, policies, procedures, and other documents affecting the regulation of generic drug products. Collaborates with all FDA offices supporting the generic drug program to ensure timely implementation and fulfillment of FDA’s commitments pursuant to the Generic Drug User Fee Amendments (GDUFA). Ensures consistency across CDER policies and procedures affecting regulation of generic drug products, including development and implementation of policy solutions to address lifecycle management matters. Provides clarity to internal and external stakeholders on generic drug regulatory policies and procedures, including development and clearance of responses to inquiries from the executive branch, Congress, media, industry, and foreign governments."

It should not escape anyone's notice that this is an FDA team pivotal to the development, production and distribution of generic drugs (and biosimilars) across the United States.

People could be forgiven for feeling relieved to see the team reinstated.

More to come on developments at FDA folks—stay tuned and feel encouraged—Gold Standard FDA is on the march!