CBER Director Notes FY 2018 Accomplishments, Including Approval of Two Gene Therapies

Looking back over the past fiscal year, Peter Marks, director for Center For Biologics Evaluation And Research (CBER) at the U.S. Food and Drug Administration (FDA), said 2018 was productive and exciting as the FDA department continued to support the advancement of 21st-century medicine.

In a Fiscal Year 2018 annual report, Marks provided some of the key highlights for the CBER, a division of the FDA that regulates biological products for human use, pointed to the approval of two gene therapies as key accomplishments. Marks said that the CBER advanced public health in FY 2018 (and beyond) by exceeding expectations in its scientific and regulatory review of new products. Marks highlights several of the products, including the approval of two new gene therapies.

In October 2017, the FDA approved Kite Pharma’s (now Gilead Sciences) Yescarta, the second CAR-T therapy approved in the United States. Yescarta was approved as the first chimeric antigen receptor T cell (CAR-T) therapy for relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. Marks also noted the December 2017 approval of Spark Therapeutics’ Luxturna, gene therapy for a rare, genetic form of blindness. The approval of Luxturna was a first, as it was the first time the FDA has approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to the approvals of the two gene therapies, Marks also said there were several other medication approvals that were noteworthy, including Dynavax’s Heplisav-B, the first new hepatitis B vaccine approved in the U.S. in more than 25 years. The FDA approved Heplisav-B in November 2017. It was the third time that the drug had been before the regulatory agency. Marks noted that the vaccine was approved to prevent infection caused by all known subtypes of hepatitis B virus in adults 18 through 70 years of age.

Another noted approval was GlaxoSmithKline’s shingles vaccine, Shingrix, as well as GSK’s Fluarix Quadrivalent, an influenza vaccine approved for children as young as six months old.

Marks said the FY 2018 report highlights accomplishments of the FDA department and reflects the agency’s “responses to new and ongoing scientific and regulatory challenges, such as those posed by advanced therapies, emerging infectious diseases, and threats to the blood supply.” The report, Marks said, demonstrates CBER’s “continuing dedication” to its mission of “meeting the challenge of improving public health, nationally and globally.”

“It also reflects how we are carefully focusing our own research and stakeholder outreach so they support our efforts to meet those challenges,” Marks said in a statement.

Not only did the regulatory agency approve cutting-edge treatments, but Marks also pointed to several other accomplishments, such as the approval of new tests for the direct detection of Zika Virus RNA in human plasma from individual donors. Also, another highlight was CBER’s granting of an emergency use authorization to the Department of Defense to enable the use of Freeze-Dried Plasma to treat hemorrhage or coagulopathy of U.S. military personnel injured during military combat when plasma is not available or its use is not practical.

CBER also continued to move forward in addressing the 21st Century Cure Act, including patient experience in regulatory decision making, providing grants to research institutions, drafting guidances for regenerative medicine and contributing to a guidance on antibacterial and antifungal drug