FDA Aims to Facilitate Gene Therapy Development for Hemophilia, Rare Diseases


The FDA has issued disease-specific draft guidances for the development of gene therapies for certain conditions.

Significant progress has been made in gene therapy research and development over the years, with promise to treat and cure some of the most complex diseases.

In a statement, FDA Commissioner Scott Gottlieb, MD, announced the agency’s latest efforts to speed up the path to approval for gene therapies, specifically for certain specialty and rare diseases.

To help expedite advancement in the field, the FDA has released a new policy framework for the development, review, and approval of gene therapies to address the challenges of bringing these drugs to market.

According to Dr Gottlieb, the latest framework includes 6 scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how the agency is going to advance the field of gene therapy while making sure new products meet the agency’s standards.

In 2017, the FDA approved the first gene therapy, tisagenlecleucel (Kymriah) for refractory B-cell precursor acute lymphoblastic leukemia, followed by the approvals of axicabtagene ciloleucel (Yescarta) for diffuse large B-cell lymphoma and voretigene neparvovec-rzyl (Luxturna) for the treatment of a rare vision loss disease.

Gene therapies are being studied as treatments for many different diseases, including autoimmune conditions, heart disease, cancer, and HIV/AIDS. A number of gene therapies in the pipeline have produced positive results in clinical trials treating diseases for which there are significant unmet needs.

“As we develop this evidence-based framework, we’re going to have to modernize how we approach certain aspects of these products in order to make sure our approach is tailored to the unique challenges created by these new platforms,” Dr Gottlieb noted in the statement.

Related Coverage: Newly Discovered Structure Could Boost Gene Therapy Efficacy

Among the draft guidance documents are the first 3 disease-specific guidances that the FDA is issuing for gene therapy products:

1. Human Gene Therapy for Hemophilia: Once finalized, this guidance will focus on gene therapy products that are targeted for the treatment of hemophilia.

2. Human Gene Therapy for Retinal Disorders: This guidance will assist those developing gene therapy products for a wide variety of retinal disorders affecting both adult and pediatric patients.

3. Human Gene Therapy for Rare Diseases: The guidance will provide recommendations on preclinical, manufacturing, and clinical trial design for all phases of the clinical development program for these types of gene therapies.

“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 durability of response, which often can’t be answered in any reasonably sized pre-market trial,” Dr Gottlieb said. “For some of these products, we may need to accept a level of uncertainty around these questions at the time of approval.”

Additionally, the FDA updated 3 existing guidances that address manufacturing issues related to gene therapy, reflecting input from various stakeholders.

The recommendations can be found in the draft guidances:

· Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)

· Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up

· Long-Term Follow-Up After Administration of Human Gene Therapy Products

“Effective tools for reliable post-market follow up, such as required post-market clinical trials, are going to be one key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments,” Dr Gottlieb stated.

According to the statement, the documents are being issued in draft form so that the agency can solicit public input on the new policies prior to finalization.


Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies [FDA statement]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613026.htm. Accessed July 11, 2018.

Related Videos
male pharmacist using digital tablet during inventory in pharmacy | Image Credit: sofiko14 - stock.adobe.com
Pharmacist holding medicine box in pharmacy drugstore. | Image Credit: I Viewfinder - stock.adobe.com
Pharmacy Drugstore Checkout Cashier Counter | Image Credit: Gorodenkoff - stock.adobe.com
Medicine tablets on counting tray with counting spatula at pharmacy | Image Credit: sutlafk - stock.adobe.com
Capsules medicine and white medicine bottles on table | Image Credit: Satawat - stock.adobe.com
Human cell or Embryonic stem cell microscope background | Image Credit: Anusorn - stock.adobe.com
Concept of health care, pharmaceutical business, drug prices, pharmacy, medicine and economics | Image Credit: Oleg - stock.adobe.com
Biosimilar pharmaceutical drug bottle on blue background. | Image Credit: Carl - stock.adobe.com
Pharmaceutical manufacture background with glass bottles with clear liquid on automatic conveyor line. | Image Credit: wacomka - stock.adobe.com
Bottle and scattered pills on color background, top view | Image Credit: New Africa - stock.adobe.com
© 2024 MJH Life Sciences

All rights reserved.