PDUFA VII will help clarify CMC requirements for cell and gene therapy firms according to the Alliance for Regenerative Medicine (ARM).

Gareth Macdonald

November 29, 2022

3 Min Read
Cell and gene therapy regulations moving in right direction says ARM
Image: DepositPhotos/ filmfoto

PDUFA VII will help clarify CMC requirements for cell and gene therapy firms according to the Alliance for Regenerative Medicine (ARM), which says regulations around such products are moving in the right direction.

The Prescription Drug User Fee Act (PDUFA) VII – legislation that enables the US FDA to collect use fees from drug developers and strengthens the agency’s review capabilities for small and large molecule drugs and advanced therapeutics – covers the fiscal years 2023 to 2027.

Cell and gene therapy is big focus in PDUFA VIII according to Michael Lemke, ARM vice president of science and industry affairs, who expressed support at the recent Cell and Gene Therapy Manufacturing and Commercialization Conference.


Image: DepositPhotos/

“The most immediate and urgent issue right now is the reauthorization of the Prescription Drug User Fee Act. For those of you who aren’t familiar that’s the law in the United States that allows FDA to charge sponsors for their submissions. And it’s all divisions of FDA.

“But also in exchange, the FDA commits to certain metrics in terms of response times for submissions, and whatever it is, this Act has to be periodically pre-authorized, and this is the seventh time that’s been done. And whenever that happens, there’s a negotiation and FDA agrees to certain initiatives and performance criteria in exchange for printing for reauthorization.”

And this time around the FDA’s Center for Biologics Evaluation and Research (CBER) – which has oversight of cell and gene therapies – is set for more personnel (FTEs or full time equivalents) Lemke said.

“So in PDUFA VII, in particular, we’re looking at, I think, a vital increase in resources to help CBER to deal with the wave of cell and gene therapy submissions. So they will receive over 100 New FTEs in fiscal year 2023. And a significant number of additional FTEs in coming years.”

CMC clarification

The new Act is also likely to clarify some longstanding issues with regard to chemistry manufacturing and control – CMC – requirements for cell and gene therapies according to Lemka, who predicted the proposed legislation would go on to win further approval

“It [PDUFA VII] also contains some important CMC provisions. Last week, it was announced that PDUFA VII will be rolled into another bill to approve spend and prevent government shutdown. The bill is pretty clean, and we’re confident that it’s going to pass in the next couple of weeks.”

On the other side of the Atlantic, the regulatory landscape for cell and gene therapy manufacturing is also evolving. In October the European Commissioned outlined plans to try and secure supply chains as part of the development of the wider Pharmaceutical Strategy for Europe that began in 2020.

And the ARM is lobbying on behalf of cell and gene therapy developers according to Lemke, who said, “In Europe, we are working on ensuring that the current revision of the EU pharmaceuticals act recognizes the unique nature of cell and gene therapies and promotes patient access.”

Lemka concluded by saying that “I believe that manufacturing is being optimized and this will continue to be a trend. And we look forward to an improved policy and regulatory environment. And particularly some clarification on some CMC issues such as potency assay development.

“And what all these things have in common is that they will increase access for patients and allow us to get more of these therapies to the patients that need them.”

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