6:30 AM
Jul 11, 2018
 |  BC Extra  |  Politics & Policy

FDA releases gene therapy guidance documents

Editor's Note: This article was updated on Jul 11, 2018 at 7:47 AM PDT

FDA Wednesday released six draft guidance documents on gene therapy: three advising companies on how to use the technology to treat certain diseases, two about manufacturing and one on the design of long-term follow-up studies.

The disease-specific draft guidance documents provide advice about developing gene therapies for hemophilia, retinal disorders and rare diseases.

Rare diseases are attractive targets for gene therapy because 80% are the result of single-gene defects, and the lack of effective therapies represents a large unmet need, FDA Commissioner Scott Gottlieb said in a statement.

The hemophilia guidance includes recommendations on the use of factor activity levels as a surrogate endpoint for accelerated approval.

One of the draft manufacturing guidance documents covers testing of retroviral vector-based gene therapies for replication competent retrovirus during manufacturing and patient follow-up. The other provides recommendations regarding CMC information in IND submissions for human gene therapies.

The new guidance documents follow three FDA approvals of gene therapies over the past year: retinal dystrophy therapy Luxturna voretigene neparvovec-rzyl from Spark Therapeutics Inc. (NASDAQ:ONCE) and CAR T cancer therapies Kymriah tisagenlecleucel from Novartis AG (NYSE:NVS; SIX:NOVN) and Yescarta axicabtagene ciloleucel from Gilead Sciences Inc (NASDAQ:GILD).

Gottlieb noted differences between regulating gene therapies and other kinds of biopharmaceuticals. “In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial,” he wrote.

These challenges mean that for some gene therapy products, “we may need to accept some level of uncertainty around these questions at the time of approval," he wrote.

Addressing uncertainty about long-term durability and safety through postmarket follow-up studies will be critical to advancing gene therapy, Gottlieb said.

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