Regulatory Updates

The American Society of Gene & Cell Therapy monitors regulatory activity in the United States that may affect the gene and cell therapy field. The information on this page provides ASGCT members and stakeholders with updates on the release of FDA guidance documents related to the field, as well as other relevant regulatory developments.

FDA Guidance Documents

Regulatory News

FDA Guidance Documents

FDA Draft Guidance on Epidermolysis Bullosa Includes Gene Therapy Trial Requirements

Updated June 20, 2018

The FDA on June 18 issued a draft guidance document, Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations. The guidance, which has a 60-day comment period, indicates that studies involving drug products intended for gene therapy should include assessments related to lentivirus vector-based risks and long-term follow-up. It also references prior guidance documents on the design of early-phase clinical trials of cellular and gene therapy products and observing subjects for delayed adverse events in gene therapy clinical trials.

Epidermolysis Bullosa:
Developing Drugs for Treatment
of Cutaneous Manifestations

FDA Issues 5 Neurological Disorder Treatment Development Documents

Updated February 19, 2018

The FDA on February 15 issued five new guidance documents on the development of treatments for neurological disorders. Two of these guidance documents address diseases that have gene therapies in clinical trials—a final guidance document on X-linked Duchenne muscular dystrophy (DMD), related dystrophinopathies and a draft guidance on amyotrophic lateral sclerosis (ALS), and early Alzheimer's disease:

Duchenne Muscular Dystrophy and Related
Dystrophinopathies - Developing Drugs for Treatment

Regulatory News

FDA Committed to Advancing Progress in Gene Therapy

Updated: June 8, 2018

The FDA announcement on June 6 of its new efforts to advance biotechnology innovation is the most recent indication of its support of the development of novel technology, including genome editing. The FDA’s new Biotech Working Group aims to ensure a flexible regulatory framework for evaluating the safety of products with indirect public benefit, namely plant and animal biotechnology innovation, including genome editing for uses such as improving the health of food-producing animals by reducing their susceptibility to diseases, and developing disease-resistant plants, which can positively affect consumers through better yields and healthier nutrient profiles.

As reported previously on ASGCT’s Twitter feed, FDA Commissioner Scott Gottlieb reported on the FDA’s intent to advance progress of gene therapy that directly affects human disease in a speech on May 22 at the Legislative Fly-In hosted by the Alliance for Regenerative Medicine in collaboration with ASGCT. The FDA intends to release a suite of draft guidance documents articulating its framework for the manufacturing and clinical development of gene therapy products beginning with a guidance on gene therapy for hemophilia, expected to be released the middle to end of this month.

The guidance documents will identify potential accelerated approval endpoints, such as the potential for factor production to be sufficient in some cases as a surrogate measure of benefit for hemophilia, with the ability to identify reduction in bleeding rates in post-market research. Other highlights of the speech include that the FDA has an internal initiative to improve the yield of cell lines used to produce gene therapy vectors, and the agency is taking steps to investigate the application of advanced manufacturing technologies, such as continuous manufacturing. These efforts are an attempt by the FDA to provide tools to expedite the development process for potentially life-altering gene therapies to treat multiple diseases with unmet medical need.

FDA Announces Pilot for Orphan Designation Requests

Updated: March 7, 2018

FDA Commissioner Scott Gottlieb, M.D. announced last week new actions the FDA is taking to support and expedite development of rare disease products, including a new pilot for more efficient orphan designation requests. A new fillable form will ease the submission process for sponsors completing designation requests, and increase the efficiency of FDA review. The FDA also launched a new online tutorial to guide sponsors through the orphan designation process.

ASGCT Submits Comments on FDA Draft Guidance on Regenerative Medicine Expedited Programs

Updated February 19, 2018

ASGCT leadership submitted comments to the FDA last week on the draft guidance titled, “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.” ASGCT expressed appreciation for guidance on these programs, especially on the Regenerative Medicine Advanced Therapy (RMAT) designation and the inclusion of certain gene therapies in the eligibility for consideration for RMAT designation. The guidance identifies gene therapies, including genetically modified cells, which lead to a durable modification of cells or tissues, as eligible for RMAT designation. A primary recommendation of ASGCT is to expand the eligibility for RMAT designation to all gene therapies to encourage the development of such therapies, which hold the potential to provide treatment for significant unmet need for multiple diseases.

FDA’s Rachel Sherman Addresses Right to Try, FDA Communication, and More

Updated February 19, 2018

Last week FDA Principle Deputy Commissioner Rachel Sherman spoke at the BIO CEO & Investor Conference in New York about a variety of topics of interest to the gene and cell therapy communities, including Right to Try legislation and enhanced communication with the FDA during drug development. This article provides an overview of the issues Dr. Sherman addressed.

“Our primary concern—really our only concern—in this bill is that patients be protected," Sherman said of Right to Try proposals. "In the initial iterations of this bill, there was no stipulation about manufacturing. You could be making [a drug] in your bathtub at home and you could be selling it.”

Government Relations Publications

Read recent ASGCT publications including the white paper on gene editing and our response to the National Center for Advancing Translational Sciences request for information.

Legislative Updates

Learn about recent legislative issues that ASGCT is monitoring or taking action on and read more about regularoty changes that affect the field of gene and cell therapy. 

Recent Collaborative Government Affairs Actions

Learn about ASGCT's most recent legislative actions and join our collaborative efforts. 

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