FDA's advanced therapies office provides more clarity on gene therapy CMC considerations
29 Sep 2022
Gene Therapy
As the Office of Tissue and Advanced Therapies (OTAT) transforms into the Office of Therapeutic Products (OTP), with new user fee funds and “super office” status, the department focused on cell and gene therapies also opened its doors to a town hall Thursday offering clarification on guidance and regulations for manufacturers.
Some of the major concerns from manufacturers were the CMC considerations between first-in-human studies and late-phase studies supporting a marketing approval.
Denise Gavin, chief of the gene therapy branch at the FDA, noted that the main concern for Phase I studies is to ensure the safety of participants, and expectations were published in previous guidance in 2020.
Preview
Source: Endpts
Kimberly Schultz
“For first in human studies, the materials used to make the product should be qualified as suitable for clinical use. We also want to make sure that the clinical materials are comparable to the preclinical material so that we can ensure that the preclinical safety data is applicable. The product and the process should be sufficiently described in your IND and supported with data so that we can assess product safety. And as clinical development progresses, the expectation is that product characterization and controls need to increase at the same pace,” Gavin said.
For products heading into Phase III, Kimberly Schultz, another chief in the gene therapy branch, said that while safety is of the utmost concern, later stage studies should also reflect a “planned commercial setting.” Companies should have the appropriate cGMPs in place as well.
Anna Kwilas, a gene therapy CMC team lead at the FDA, noted that when it comes to device-biologic compatibility for gene therapies, the main goal for manufacturers is to show that the device the therapy is delivered in will not affect the product’s quality.
Preview
Source: Endpts
Anna Kwilas
“So console credibility studies should be collected, [and] compatibility study should be collected with the final formulated drug product under the worst-case conditions expected in the clinical trial,” Kwilas said.
Another major concern for manufacturers was logistical challenges surrounding autologous cell-based gene therapies, especially when it comes to expectations for process characterization.
Schultz stressed that in most cases it is appropriate to show healthy donor material that is representative of the patient’s specific process, however not all attributes may be tested in products manufactured using the healthy donor material. Schultz did state that OTAT would recommend that manufacturers should evaluate the small-scale manufacturing studies that can be used to support process characterization.
When it comes to rare diseases, Gavin said FDA encourages manufacturers to think in advance:
In order to determine effectiveness, we recommend that sponsors, before they administer any product, that they establish a well-controlled manufacturing process and qualified analytical test. We also recommend strongly that you plan ahead, and consider the long-term demands of the disease indication when designing a process. Sponsors often make significant changes late in development and this introduces uncertainty in the development program as analytical comparability is not always sufficient with complex gene therapy products.
FDA's OTAT morphs into the Office of Therapeutic Products as new funds await
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