Two gene therapy approvals headline CBER’s FY 2018 report

The FDA Center for Biologics Evaluation and Research (CBER) on Wednesday published its fiscal year 2018 report, highlighting two gene therapy approvals, as well as the approvals of a hepatitis B vaccine, a vaccine to prevent shingles and a flu vaccine for children as young as six months.

Both of the gene therapy approvals occurred at the very beginning of the previous fiscal year, with CBER signing off on Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) for children and adult patients with an inherited form of vision loss in December 2017 and Gilead’s Yescarta (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma, in October 2017.

“FY 2018 was particularly exciting because of the pace of progress in cellular and gene therapies. CBER received more than 150 INDs for gene therapy products, bringing the total number of active INDs up to nearly 800 applications,” CBER Director Peter Marks said.

Peter Marks

CBER’s Office of Tissues and Advanced Therapies also released 10 new guidance documents in 2018, including six new draft guidance documents on gene therapy and a recently finalized guidance on expedited programs for regenerative medicine.

The center also launched a new program in 2018, known as INTERACT, whereby sponsors can seek a preliminary and informal consultation with the agency prior to a pre-IND meeting.

In addition, CBER met its drug and medical device user fee goals and authorized the first two donor screening tests for the direct detection of Zika virus RNA in human plasma from individual donors.

Another highlight was CBER’s granting of an emergency use authorization last July to the Department of Defense to enable the emergency use of a freeze-dried plasma product manufactured by the Centre de Transfusion Sanguine des Armées (French FDP). The product is used to treat hemorrhage or coagulopathy of US military personnel injured during combat when plasma is not available, or its use is not practical. The move follows the Pentagon’s criticism of the FDA’s sluggishness in approving the freeze-dried product, which some said other countries’ troops had used for years.

As part of the FDA’s mutual recognition agreements with Europe, CBER assessed regulators from 14 countries to ensure their inspection of medical products regulated by the center could be recognized by the FDA. And CBER said it’s continuing to assess the remaining EU member states to meet its 15 July commitment that all member states are confirmed.

What the report does not mention, however, is the growing number of Regenerative Medicine Advanced Therapy (RMAT) designation requests that the FDA is receiving. According to data as of 31 March, there have now been 97 designation requests (including 19 in the first three months of this year), of which 33 have been granted, 53 denied and five withdrawn.

CBER FY 2018 Report

First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.

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