RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > 2019 > 2 > CBER Guidance on Gene Therapies: What to Expect in 2019

CBER Guidance on Gene Therapies: What to Expect in 2019

Posted 20 February 2019 | By Zachary Brennan 

CBER Guidance on Gene Therapies: What to Expect in 2019

The US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) on Wednesday released plans for what guidance is to be expected in 2019, including six final guidance documents related to gene therapies.

The gene therapy guidance documents (draft versions were published in July) the agency expects to finalize include:
  • Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry
  • Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications
  • Human Gene Therapy for Hemophilia
  • Human Gene Therapy for Retinal Disorders
  • Human Gene Therapy for Rare Diseases
  • Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up (which was also on the 2018 guidance list).
Eight other guidance documents are expected to deal with blood products and components, including draft guidance that deals with testing for biotin interference in in vitro diagnostic devices, and another draft that will focus on revised preventive measures to reduce the possible risk of transmission of Creutzfeldt-Jakob Disease (CJD) and variant Creutzfeldt-Jakob Disease (vCJD) by blood and blood products. In addition, other guidance is expected to be finalized, including one on further testing of donations that are reactive on a screening test for antibodies to hepatitis C virus (which was also on the 2018 list).

Other guidance documents are expected to deal with standards development and their use in regulatory submissions and lot release protocol submissions to CBER in electronic format.

Guidance Documents CBER is Planning to Publish During Calendar Year 2019


© 2022 Regulatory Affairs Professionals Society.