Convergence: CBER chief discusses priorities, touts gene therapy approvals
MONTREAL – Officials at the US Food and Drug Administration (FDA) described some of their top priorities and touted the approval of 15 new gene therapies during a forum at RAPS Convergence 2023.FDA officials also described some of this year’s “notable” novel drug approvals and the progress being made toward resuming in-person meetings.
Vaccines, gene therapy, novel drugs
Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), told attendees at a RAPS health authority forum that the “top priority” for 2023 was to simplify and harmonize the labeling for COVID-19 vaccines and to get “an annual framework for post-Covid vaccines.”
This task has largely been accomplished with the September 2023 approval of an updated COVID-19 vaccine formulated to correspond more closely to the Omicron variant XBB.1.5, he said. “Thankfully this [vaccine] has been shown in multiple studies to neutralize what is currently circulating,” Marks said.
In the area of cell and gene therapies, Marks said the agency is “particularly excited” about its approval of 15 gene therapies. At the same time, he acknowledged that these therapies are now at a “critical juncture.” Obstacles remain for developing these products, including manufacturing challenges, clinical development timelines and divergent global regulatory requirements, he said.
Kevin Bugin, the deputy director of operations for the Office of New Drugs in FDA’s the Center for Drug Evaluation and Research, highlighted some “noteworthy” drug approvals this year. In March, the agency approved the first nonprescription naloxone nasal spray, and in July it approved both lecanemab for early Alzheimer’s disease and the first over-the-counter birth control pill.
Overall, FDA approved 40 novel drugs in Fiscal Year 2023, up from 37 in the prior year, Bugin said.
START pilot
FDA is also continuing its efforts to spur development of therapies for rare diseases. In September, the agency launched a new pilot program called Support for clinical Trials Advancing Rare disease Therapeutics (START). Through the pilot, sponsors can get advice and request ad hoc communications with FDA on product-specific development issues, including clinical study design, choice of control group and selecting patients for clinical trials.
The pilot is open to sponsors with products currently in clinical trials under an active Investigational New Drug (IND) application that is regulated through either CBER or CDER. Applications for the pilot will be accepted between 2 January 2024 and 1 March 2024.
Return to in-person meetings
During the question-and-answer session, regulators were asked when FDA would return to in-person meetings, specifically for the INTERACT (Initial Targeted Engagement for Regulatory Advice on CBER/CDER ProducTs) program.
“We are hoping that we have more live interactions,” Marks said. “I can’t tell you where we will accept more live meetings, but we certainly want more live meetings.”
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