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 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trials—a final guidance document on X-linked Duchenne muscular dystrophy (DMD), related dystrophinopathies and a draft guidance on amyotrophic lateral sclerosis (ALS), and early Alzheimer's disease: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

The draft guidance has a 60-day comment period:

Amyotrophic Lateral Sclerosis:
Developing Drugs for Treatment

The draft guidance has a 90-day comment period:

Early Alzheimer's Disease:
Developing Drugs for Treatment

FDA Issues Guidance Documents to Enhance Interactions With Product Developers

Updated: Decmeber 18, 2017

The FDA announced the availability of two guidance documents on December 28 that aim to facilitate early, thorough interaction between the FDA and product developers. The following final guidance supports communication between those submitting investigational new drug applications (INDs) and the FDA’s review staff at critical junctures in development, to maximize efficiency of the process:

Best Practices for Communication Between IND Sponsors
and FDA During Drug Development

The following draft guidance provides recommendations for efficient, consistent procedures for the timely and effective conduct of formal meetings between the FDA and those developing new drug or biologic products. This guidance has a 90-day comment period: 

Formal Meetings Between the Food and Drug Administration
and Sponsors or Applicants of PDUFA Products

FDA CBER Issues Draft Guidance on Standards

Updated: December 19, 2017

The FDA CBER issued a draft guidance that includes information on how sponsors can utilize standards to facilitate product development and FDA acceptance of use of standards. Comments may be submitted until March 19, 2018:

Standards Development and the Use of Standards in
Regulatory Submissions Reviewed in the Center for
Biologics Evaluation and Research

FDA Framework for Regenerative Medicine

Updated: November 16, 2017

The FDA announced a comprehensive regenerative medicine policy framework in November, outlined in two finalized guidance documents, and two draft guidance documents. The first final guidance document clarifies the determination of whether HCT/Ps are subject to FDA’s premarket review requirements:

Regulatory Considerations for Human Cell, Tissues,
and Cellular and Tissue-Based Products: Minimal
Manipulation and Homologous Use

The second final guidance clarifies the qualifications for an exception from the established regulations for HCT/Ps that are removed from and implanted in the same individual within the same surgical procedure and remain in their original form:

Same Surgical Procedure Exception: Questions and
Answers Regarding the Scope of the Exception

The first draft guidance document describes several expedited programs that are available to sponsors of regenerative medicine therapies, and information about the requirements for, and benefits of, the new RMAT designation program that was created by the 21st Century Cures Act.

The second draft guidance, published as a requirement of the 21st Century Cures Act, provides the agency’s current thinking about the evaluation of devices used in the recovery, isolation and delivery of RMATs. 

Regulatory News

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.”

ASGCT a Stakeholder at Cell Therapy Liaison Meeting

Updated: January 30, 2018

The International Society for Cellular Therapy annually holds meetings between the FDA Center for Biologics Evaluation and Research and over 20 invited stakeholder organizations. ASGCT had two representatives at the Cell Therapy Liaison Meeting in Bethesda, MD. These meetings enable the cell therapy community to discuss specific issues of concern with CBER and provide updates on recent developments in field. A meeting summary will be posted here when available.

FDA CBER Issues 2018 Guidance Agenda

Updated: January 19, 2018

The FDA Center for Biologic Evaluation and Research (CBER) issued a statement of the guidance documents that it intends to issue in 2018, which includes up to six guidance documents on gene therapy. All of the gene therapy topics would be new guidance documents, and will likely include:

  • Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-Up
  • Observing Subjects Who Received Gene Therapy Products for Delayed Adverse Events
  • Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug (IND) Applications
  • Gene Therapy for the Treatment of Hemophilia

The agency also intends to issue two additional clinical draft guidance documents on the development of gene therapy products.

FDA Orphan Products Grants Program Funding of Rare Disease Natural History Studies

Updated: October 10, 2017

For the first time, on October 7 the FDA awarded six new research grants for natural history studies in rare diseases to conduct rare disease natural history studies, through its Orphan Products Grants Program. The FDA is providing a total of $6.3 million over the next five years to fund four natural history studies and, through a partnership with NCATS Therapeutics for Rare and Neglected Diseases program, the FDA received $3.5 million to be combined with FDA funding to fund an additional two studies.

Such research can inform clinical trial development, and may lead to the use of natural history models to augment or replace placebo arms in studies of therapies for very rare diseases, for which trial recruitment can be difficult and for which withholding treatment may pose ethical concerns.

The grants support research on Friedreich’s ataxia; pregnancy and lactation-associated osteoporosis; sarcoidosis; sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease; Angelman syndrome; and myotonic dystrophy type 1 to determine biomarkers and clinical endpoints.  

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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