FACACLASSIC

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HHS - 127 - Cellular Tissue and Gene Therapies Advisory Committee - Authorized by Law

GENERAL INFORMATION

Committee Name	Cellular Tissue and Gene Therapies Advisory Committee	Agency Name	Department of Health and Human Services
Fiscal Year	2025	Committee Number	127
Original Establishment Date	11/28/1990	Committee Status	Chartered
Actual Termination Date		Committee URL	http://www.fda.gov/AdvisoryCommittees/Committee...
Actual Merged Date		Presidential Appointments*	No
New Committee This FY	No	Max Number of Members*	14
Terminated This FY	No	Designated Fed Officer Position Title*	Designated Federal Officer
Merged This FY		Designated Federal Officer Prefix	Dr.
Current Charter Date	10/28/2024	Designated Federal Officer First Name*	Cicely
Date Of Renewal Charter	10/28/2026	Designated Federal Officer Middle Name
Projected Termination Date		Designated Federal Officer Last Name*	Reese
Exempt From Renewal*	No	Designated Federal Officer Suffix
Specific Termination Authority		Designated Federal Officer Phone*	301-796-9025
Establishment Authority*	Authorized by Law	Designated Federal Officer Fax*	301-595-1307
Specific Establishment Authority*	21 U.S.C. 394	Designated Federal Officer Email*	cicely.reese@fda.hhs.gov
Effective Date Of Authority*	11/28/1990
Exempt From EO 13875 Discretionary Cmte	Not Applicable
Committee Type*	Continuing
Presidential*	No
Committee Function*	Scientific Technical Program Advisory Board

RECOMMENDATION/JUSTIFICATIONS

Agency Recommendation*	Continue
Legislation to Terminate Required	Not Applicable
Legislation Status	Not Applicable
How does cmte accomplish its purpose?*	The Committee reviews and evaluates available data relating to the safety, effectiveness, and appropriate use of human cells, human tissues, gene therapies, and xenotransplantation products which are intended for transplantation, implantation, infusion, and transfer in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair, or replacement of tissues for various conditions. On November 21, 2024, the Committee met in open session to discuss and make recommendations on supplemental biologics license application 125586/546 from AstraZeneca AB, submitted to confirm the clinical benefit of Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo), for patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. The FDA considered the recommendations of CTGTAC in its review of sBLA 125586/546. Approval for Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo) for the proposed indication was not granted.
How is membership balanced?*	Members have clinical or preclinical and product experience in the fields of cellular therapies, tissue transplantation, gene therapies, and xenotransplantation. These may include biostatistics, bioethics, hematology/oncology, human tissues and transplantation, reproductive medicine, general medicine, and various medical specialties including surgery and oncology, immunology, virology, molecular biology, cell biology, developmental biology, tumor biology, biochemistry, rDNA technology, nuclear medicine, gene therapy, infectious diseases, and cellular kinetics. One member is technically qualified and identified with consumer interests and one non-voting member represents the point of view of industry.
How frequent & relevant are cmte mtgs?*	The committee held one (1) advisory committee meeting on November 21, 2024, and one site visit, in FY 2025. It is anticipated the committee may have two (2) advisory committee meetings FY 2026.
Why advice can't be obtained elsewhere?*	Members of the Committee are drawn from academia, research, and/or clinical practice. Their advice and input lends credibility to regulatory decisions. The alternate means of obtaining this advice would involve the recruitment of large numbers of scientists on a full-time basis at maximum rates of compensation.
Why close or partially close meetings?	The committee held no closed meetings in FY 2025.
Recommendation Remarks	The Agency considered the recommendations of the Committee from the November 21, 2023 CTGTAC meeting. Approval was not granted for and granted approval for supplemental biologics license application 125586/546 from AstraZeneca AB, submitted to confirm the clinical benefit of Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo), for patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. The Committee agreed that while treatment with Andexxa appears to lead to achievement of hemostatic efficacy at 12 hours, the efficacy data provided did not clearly show a clinically meaningful benefit. The Committee found it challenging to assess the risks of Andexxa (e.g., increased rate of thrombotic events in Andexxa-treated patients) in the context of its current indication, given that ANNEXA-I only evaluated Andexxa in patients with life-threatening intracerebral hemorrhage after receiving oral anticoagulants (apixaban or rivaroxaban). The FDA issued a Complete Response Letter (CRL) denying granting full approval (i.e., conversion from accelerated to full approval) for Andexxa’s supplemental biologics license application.

PERFORMANCE MEASURES

Outcome Improvement To Health Or Safety*	Yes	Action Reorganize Priorities*	Yes
Outcome Trust In Government	Yes	Action Reallocate Resources	No
Outcome Major Policy Changes	Yes	Action Issued New Regulations	Yes
Outcome Advance In Scientific Research	Yes	Action Proposed Legislation	No
Outcome Effective Grant Making	No	Action Approved Grants Or Other Payments	No
Outcome Improved Service Delivery	No	Action Other	Yes
Outcome Increased Customer Satisfaction	Yes	Action Comment	FDA approves or chooses not to approve an investigational medical product or other regulatory decision-making.
Outcome Implement Laws/Reg Requirements	Yes	Grants Review*	No
Outcome Other	No	Number Of Grants Reviewed	0
Outcome Comment	Not Applicable	Number Of Grants Recommended	0
Cost Savings*	Unable to Determine	Dollar Value Of Grants Recommended	$0.00
Cost Savings Comment	The utilization of the Cellular, Tissue, and Gene Therapies Advisory Committee enables the Agency to obtain required and frequently scarce professional services from medical and scientific experts not otherwise available to the Agency, and to obtain the services of these experts only on an as-needed basis rather than a full-time basis. The services of the Committee resulted in advice for the improvement of the public health, for which it is difficult to assign a financial value.	Grants Review Comment	NA
Number Of Recommendations*	101	Access Contact Designated Fed. Officer*	Yes
Number Of Recommendations Comment	The Committee made 101 recommendations from FY 2003 through FY 2025.	Access Agency Website	Yes
% of Recs Fully Implemented*	84.00%	Access Committee Website	Yes
% of Recs Fully Implemented Comment	The function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, and therefore, the Agency has the option of not implementing the advice.	Access GSA FACA Website	Yes
% of Recs Partially Implemented*	10.00%	Access Publications	Yes
% of Recs Partially Implemented Comment	The function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, and therefore, the Agency has the option of not implementing the advice.	Access Other	No
Agency Feedback*	Yes	Access Comment	N/A
Agency Feedback Comment*	When appropriate, information is made available to the public. Actions related to guidance documents or other general matters issues are available publicly when implemented.	Narrative Description*	FDA's strategic priorities in responding to the public health challenges of the 21st century are to advance regulatory science and innovation; strengthen the safety and integrity of the global supply chain; strengthen compliance and enforcement activities to support public health; expand efforts to meet the needs of special populations; advance medical countermeasures and emergency preparedness; advance food safety and nutrition; promote public health by advancing the safety and effectiveness of medical products; establish an effective tobacco regulation, prevention, and control program; and manage for organizational excellence and accountability. The Cellular, Tissue, and Gene Therapies Advisory Committee supports FDA's mission and strategic action by reviewing and evaluating available data relating to the safety, effectiveness, and appropriate use of human cells, human tissues, gene therapies, and xenotransplantation products which are intended for transplantation, implantation, infusion, and transfer in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair, or replacement of tissues for various conditions. The Committee also considers the quality and relevance of FDA's research program, which provides scientific support for the regulation of these products. The Committee supports FDA's mission by using science-based risk management in all its activities. The Committee recommendations provide the most health promotion and protection at the least cost to the public. This Committee assists the Agency in ensuring timely, high-quality, cost-effective processes for review of new technologies/pre-market submissions, effective communication and working relationships with stakeholders to enhance U.S. and global health outcomes, accurate analysis of risks associated with medical products, facilitation of the development and availability of medical countermeasures to limit the effects of a terrorist attack on the civilian and military populations, and protection of the safety and security of biologics (gene therapy, human tissues, and cellular therapies) – all key components of FDA's strategic plan objectives.

COSTS

1. Payments to Non-Federal Members*		1. Est Paymnts to Non-Fed Membrs Nxt FY*
2. Payments to Federal Members*		2. Est. Payments to Fed Members Next FY*
3. Payments to Federal Staff*		3. Estimated Payments to Federal Staff*
4. Payments to Consultants*		4. Est. Payments to Consultants Next FY*
5. Travel Reimb. For Non-Federal Membrs*		5. Est Travel Reimb Non-Fed Membr nxtFY*
6. Travel Reimb. For Federal Members*		6. Est Travel Reimb For Fed Members*
7. Travel Reimb. For Federal Staff*		7. Est. Travel Reimb to Fed Staf Nxt FY*
8. Travel Reimb. For Consultants*		8. Est Travel Reimb to Consltnts Nxt FY*
10. Other Costs		10. Est. Other Costs Next FY*
11. Total Costs	$0.00	11. Est. Total Next FY*	$0.00
Date Cost Last Modified	3/14/2025 9:25 AM	Est. Fed Staff Support Next FY*
Federal Staff Support (FTE)*		Est Cost Remarks
Cost Remarks

Interest Areas

Category Area Food and Drugs Food and Drugs Health Treatment Medicine Diseases

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