Cell and gene therapies: FDA official on COVID-19 impact

Regulatory NewsRegulatory News | 08 June 2020 |  By 

Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US Food and Drug Administration (FDA) official discussed the impact of the coronavirus disease (COVID-19) pandemic on the agency’s work related to cell and gene therapies.
“Before the pandemic, we were seeing a huge escalation in the workload that we have to make adjustments for,” said Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the Center for Biologics Evaluation and Research (CBER), adding the number of investigational new drug applications (INDs) for gene therapies his office receives has tripled in the last three years.
While the pandemic has not resulted in an increase in applications for gene therapies, Bryan said his office has been busy with an increase in interest in using cell therapies and intravenous immunoglobulin (IVIG) to treat COVID-19.
“Our clinical team particularly has been stretched trying to deal with the interest for use of immunoglobulin products and the use of cellular products for the treatment of patients who are seriously ill with COVID-19, as well as some cases of thinking about using these products for prophylaxis,” he said.
However, even with the pressing need for new treatments for COVID-19, Bryan stressed that well-designed clinical trials are essential in the long run. “At the end of the day we’ve got to come through this pandemic knowing which products help patients and which ones don’t. In some cases, we see clinical trials that are small and uncontrolled and really aren’t going to move us forward much. But then we see some sponsors who are doing well-designed clinical trials that hopefully will help us to find treatments for this disease.”
As with other offices at FDA, Bryan said his team is prioritizing work related to the pandemic and said that industry can expect some delays in terms of responses, meetings and new guidance.
“You may find that we’re a little bit slower in responding and we may give written responses instead of a [teleconference]. Ultimately the meeting still gets scheduled and we still respond, it just might not be as quick,” he said.
Snehal Naik, associate director, global regulatory affairs, regenerative medicines and advanced therapies at Janssen, said she is looking forward to CBER’s upcoming draft guidance on genome editing, as well as other disease-specific guidances following FDA’s finalization of six gene therapy guidances and issuance of a draft guidance earlier this year. (RELATED: FDA Finalizes 6 Gene Therapy Guidances, Unveils a New Draft, Regulatory Focus 28 January 2020).
“It’s been so valuable to have the ones around retinal disease and hemophilia, and next on our wish list, which was reflected on the guidance agenda, was neurodegenerative diseases because we’re starting to see an increasing number of trials of gene therapy applications there as well,” Naik said.
Bryan responded by cautioning that those guidances will likely be delayed due to the pandemic. While not giving a specific timeline, he said that, “Some of these guidances might not come out as quickly as we had hoped, but they are still on the docket and we’re still working on them.”
Additionally, Bryan stressed that sponsors, especially academic ones, or those less experienced with commercial manufacturing, should put more emphasis on manufacturing early on in development.
“We’re seeing so much delay in bringing products to market because folks just are not paying attention to the manufacturing issues,” he said, adding that in some cases manufacturing issues are causing delays upward of one year.
Bryan also tempered expectations on the prospect of greater international harmonization for cell and gene therapies, saying that “harmonization is probably a ways away.”


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