FDA Official: New “Playbook” Needed for CMC Reviews of Gene Therapy Products

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analytics so that we can gain product and process understanding so that we can facilitate rapid changes that we know will not negatively impact the health of the patients.”


Walker said a constriction point for her is not keeping up to date with current technologies. She said that a technology platform developed today may be outdated tomorrow. The rate of change of innovation is now every two or three years, she said.

That mirrors the rate of the device cycles that Marks mentioned, lending further credence to the idea that cell and gene therapies should be reviewed similarly.

Walker said that “the technology that Kymriah has been based on has been eclipsed. It took three years for start up to approval and now no one is using the same technology as the basis for their platform. The technology and the state of the art is advancing very rapidly. This is a challenge for regulators. They need to understand we can be early adopters of these technologies without changing the product.”

Kymriah was the first gene therapy product approved to treat B-cell acute lymphoblastic leukemia (ALL) and diffuse B-cell lymphoma (DLBCL). The product used spherical beads to isolate, activate and expand T-cells. After the cells are modified, they are infused back into the patient. The FDA approved the drug in August 2017 (Also see “FDA’s NDA And BLA Approvals: Kymriah, Vabomere, Cyltezo” – Pink Sheet, 1 Sep, 2017.) and the EU approved it in June 2018. (Also see “First CAR T-Cell Therapies OK’d In EU: Novartis’s Kymriah And Kite’s Yescarta” – Pink Sheet, 29 Jun, 2018.)

Walker said there also needs to be flexibility from regulators to allow new technologies. “The technology is advancing very rapidly, and that is another challenge for regulators. To understand where they can have flexibility.”


Michael Paglia, senior VP of CMC operations for ElevateBio, said that his main constriction point has to do with “staff and talent and supply chain and the cost of goods and quality and access to capacity.”

To address the capacity challenges, the company came up with a model of funding multiple start-ups and to utilize the same R&D and manufacturing facility, rather than individual companies whose cell and gene therapy R&D is slowed by the need to build their own lab and production spaces. (Also see “ElevateBio Brings Centralized Model To Cell And Gene Therapy” – Scrip, 13 May, 2019.)

“We took the approach to build our own and to build an integrated research to support out cell and gene therapies.”

The company in July 2019 announced a partnership with Massachusetts General Hospital. Under the agreement, which runs for 10 years, MGH has access to ElevateBio’s research, process development and manufacturing facility in Waltham, MA, for development and production of cell and gene therapies.

Paglia said that there are now more skilled employees compared to “seven or 10 year ago,” but that it is still challenging to find talent. “We are fortunate to have experienced staff. It is necessary to put procedures in place to have rigorous training. Training is very important, and we are involved with local universities as well to give them an idea of if you come out of this how do you get into cell and gene therapy.”

This article was first published in the Pink Sheet on June 18, 2020.

Image: iStock/IvelinRadkov

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