Marks talks OTP staffing, gene therapies, and more

Editor's Note: A previous version of this story incorrectly reported that FDA had chosen three applicants to particpate in the START pilot.

WASHINGTON – The US Food and Drug Administration’s (FDA) top biologics official expects that 2024 will be a “breakout year” for gene therapy approvals, as a new number of new initiatives take hold to promote the development of these products.

Speaking at the 2024 BioPharma Congress on 26 February, Peter Marks, director the Center for Biologics Evaluation and Research (CBER), said that the recently established Office of Therapeutic Products (OTP) is now 75-80 percent staffed.

He also discussed some of the opportunities and some of the agency’s methods for promoting the development of cell and gene therapies with moderator Michael McCaughan of Prevision Policy.

“What we are trying to accomplish this year is taking a number of the developments that have started to accelerate over the past year, then taking what we learn and apply it. That means applying the accelerated approval provision and optimizing communications with sponsors and increasing our global cooperation with other regulators.

These initiatives include FDAs recently finalized guidance stating that the accelerated approval pathway may be used for sponsors developing gene therapy product that incorporate genome editing (GE) of human somatic cells and the center’s recently announced Support for clinical Trials Advancing Rare disease Therapeutics (START) communications pilot for rare diseases. (RELATED: FDA: Genome editing therapies may use accelerated approval pathway, Regulatory Focus 30 January 2024)

OTP staffing

In December 2021, when the former Office of Tissues and Advanced Therapies (OTAT) had problems recruiting staff, OTAT’s then-director Wilson Bryan said a growing workload and insufficient staffing prompted the office to institute new processes to expedite its reviews of new cell and gene therapies. (RELATED: Gene therapy developers can expect less hand-holding from OTAT, Regulatory Focus 10 December 2021)

He noted though that while OTP is now nearly fully staffed, Marks said the agency is still “actively recruiting” personnel.

When asked to comment on how long it takes to get reviewers full trained, Marks said this varies, and that it may take less training for a physician with clinical experience than someone with a background in chemistry, manufacturing, and controls (CMC) who is pursuing a PhD.

START pilot

When asked about the START pilot, Marks said “we have heard a lot of interest, yet I would have expected more applications.” He expects to get more applications on the upcoming Rare Disease Day. Three applicants will be selected to participate. The application deadline will close on 1 March.

CBER launched the START pilot in September 2023; the program enables sponsors to get advice and request ad hoc communications from reviewers on product-specific development issues, including clinical study design, choice of control group, and clinical trial patient selection. The goal of the pilot is to accelerate the pace of development of treatments for rare diseases that untreated can likely to lead to significant disability or death. (RELATED: Convergence: CBER chief discusses priorities, touts gene therapy approvals, Regulatory Focus 5 October 2023).

He was also asked to address the milestone approval of last year’s first CRISPER-Cas9-edited cell therapy, Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) for sickle-cell disease and whether CRISPR therapies should be considered differently than gene therapies.

Marks said that these products are considered gene therapies and are not considered medical devices.

He further noted that the CRISPR field is evolving at a rapid pace since first described in the literature 12 years ago. “It is remarkable, we are seeing a new generation of CRISPR fine editors.”

Marks further observed that these technologies can be the key to producing treatments on a larger scale and that “current gene therapies based on AAV [adeno-associated viruses] cannot be made to mass scale.”

Platform guidance forthcoming

In other areas, Marks said that a draft guidance on the platform technology designation program would be forthcoming, when asked to address the status of the document.

These technologies received a boost through the omnibus spending bill approved by Congress in December 2022, which directed FDA to establish a platform designation for drugs or biological products that have technologies that can be incorporated in or used by more than one drug or biological product. (RELATED: Omnibus brings new advanced manufacturing programs to FDA, Regulatory Focus 11 January 2023).

BioPharma Congress

Marks talks OTP staffing, gene therapies, and more

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