Replimune's Tudriqev (vusolimogene oderparepvec-wtpg) was approved on August 6, 2026 under accelerated approval, in combination with nivolumab, for adults with unresectable advanced cutaneous melanoma that progressed on a PD-1 based regimen. Approval was based on objective response rate and duration of response in the single-arm IGNYTE study. The company reports an objective response rate of 24.2% and a median duration of response of 14.1 months in 91 efficacy-evaluable patients. The ongoing Phase 3 IGNYTE-3 trial is the confirmatory study. This was the third submission and followed the July 30 CTGTAC vote.
Ultragenyx's Genglycos (pariglasgene brecaparvovec-opnr) was approved on August 19, 2026 under accelerated approval, as an adjunct to nutritional management to reduce daily cornstarch intake in patients 8 years and older with glycogen storage disease type Ia (GSDIa), based on a 31% mean reduction in cornstarch intake versus placebo at 48 weeks.
Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) was approved on September 17, 2026 as the first treatment for pediatric patients with Sanfilippo syndrome type A (MPS IIIA). It is a one-time intravenous AAV9 gene therapy. Supporting data came from an open-label, single-arm study in children aged 2 to 5 compared with an untreated historical cohort.
Vertex’s Casgevy label expansion to patients 2 years and older with sickle cell disease or transfusion-dependent beta thalassemia was announced July 1, 2026. FDA reports it came 53 days after filing and was the eighth approval under the Commissioner's National Priority Voucher (CNPV) program.
Deramiocel (Capricor) review was extended. After the advisory committee, Capricor submitted 24-month open-label extension data from the Phase 3 HOPE-3 study, additional robustness analyses, and a refined indication focused on upper limb function. FDA classified the submission as a major amendment, which moved the action date from August 22 to November 22, 2026 (company announcement, August 24).
Tabelecleucel (Pierre Fabre, with Atara) was resubmitted on or about September 30, 2026 for EBV-positive post-transplant lymphoproliferative disease, following a second Complete Response Letter in January 2026.
For the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:
Patients aged 12 years and older with:
Hemophilia B (congenital Factor IX deficiency) adults with Hemophilia B (congenital Factor IX deficiency) who:
**This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
List does not include HPC, Cord Blood products for use in unrelated donor hematopoietic progenitor cell transplantation procedures.
List also does not include previously approved products that are no longer being marketed.
The following information on BLAs currently under US FDA review was obtained from company press releases.
CTGTAC — Deramiocel / Capricor (July 29, 2026). The Cellular, Tissue, and Gene Therapies Advisory Committee will meet to review BLA 125842 for deramiocel (allogeneic cardiosphere-derived cells; Capricor) for cardiomyopathy in Duchenne muscular dystrophy. PDUFA date: August 22, 2026. The meeting will be webcast publicly.
CTGTAC — Vusolimogene Oderparepvec / Replimune (July 30, 2026). The Committee will review BLA 125827 for vusolimogene oderparepvec (Replimune) in combination with nivolumab for advanced melanoma in patients who have previously received anti-PD-1 therapy.
FDA Rare Disease Framework Initiative Public Meeting (October 28, 2026). FDA announced that it will hold a public meeting at its White Oak campus with a virtual participation option.
FDA Scientific Public Workshop: Next-Generation Sequencing for Adventitious Agent Detection in Biologics (September 23, 2026). OTP and the Office of Vaccines Research and Review scheduled this virtual workshop covering FDA perspectives, CRO implementation of NGS, NGS for adventitious virus detection, and non-viral agents.
PDUFA VIII public meeting (September 16, 2026). FDA held a hybrid public meeting on the proposed PDUFA VIII commitment letter. Written comments are due October 16, 2026 (docket FDA-2026-N-8163).
OTP Town Hall: BLA Submission Best Practices, transcript now posted. The June 4 town hall highlighted in the July issue has a transcript on FDA's site.
OTP Town Hall — BLA Submission Best Practices (June 4, 2026). OTP's Office of Review Management & Regulatory Review (ORMRR) hosted a virtual town hall on BLA readiness for CGT products, covering how to request pre-BLA meetings, best practices for submission packages, and available FDA tools.
OTP/ARM Workshop — Advancing Pediatric CGT Clinical Trials (April 9, 2026). CBER/OTP and the Alliance for Regenerative Medicine co-hosted a hybrid workshop at FDA's White Oak campus addressing clinical trial design for CGT in pediatric populations, with emphasis on conditions where earlier intervention improves outcomes.
Individualized Therapies On The Rise- November 20, 2025
OTP Town Hall: Gene Therapy Manufacturing CMC and Facility Readiness for BLAs and Post-licensure Changes- October 22, 2025
For CGT
Also applicable to CGT
Journal publications and webposts from FDA:
Flexible Requirements for Cell and Gene Therapies to Advance Innovation
FDA Public Meeting: Onshoring Manufacturing of Drugs and Biological Products – September 30, 2025
Lessons Learned From the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program - September 10, 2025
On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations – September 3, 2025
FDA Roundtable on Cell and Gene Therapy June 5, 2025
OTP Town Hall: Best Practices for Regulatory Interactions with OTP – December 12, 2024
Meeting 2: Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases –
December 4, 2024
Workshop on Integration Site Analysis During Long Term Follow-Up for Gene Therapies with Integrating Viral Vectors –
November 19, 2024
Finding Your Support Team While Participating in a Clinical Trial –
October 30, 2024
Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub
October 16, 2024-recording not yet available
OTP Town Hall: Cell Therapy CMC Readiness for Late-Stage INDs
September 5, 2024- recording available
FDA Public Workshop on Best Practices for Meeting Management
July 22, 2024- recording and event materials available
The following links to past Meeting Materials include access to the recorded video link, transcript, or slides.
OTP Town Hall: CMC Readiness for Gene Therapy BLAs
June 4, 2024
Nonclinical Assessment of Cell and Gene Therapy Products
August 30, 2023
OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 2023
June 8, 2023
OTP Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls – April 2023
April 25, 2023
Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products
April 27, 2023
Clinical Trials: The Patient Experience
April 13, 2023
FDA CBER Office of Therapeutic Products (OTP) Advanced Manufacturing and Analytical Technologies (AMAT) for Regenerative Medicine Therapies (RMT) Workshop
March 14, 2023
OTAT Town Hall: Clinical Development of Gene Therapy Products for Rare Diseases
February 7, 2023
Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products
December 16, 2022
OTAT Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls
December 7, 2022
FDA/PQRI Workshop on the Regulatory Framework for Distributed and Point of Care Pharmaceutical Manufacturing: An Opportunity for DM/POC Stakeholder Engagement
November 14 - 16, 2022
FDA CBER OTAT Patient-Focused Drug Development Listening Meeting — Patient Perspectives on Gene Therapy Products
November 15, 2022
Natural History Studies to Support Regenerative Medicine: A How-To Webinar
October 27, 2022
OTAT Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls
September 29, 2022
2022 Center for Biologics Evaluation and Research (CBER) Science Symposium
September 20-22, 2022
Annual Patient Engagement & Regenerative Medicine Meeting 2022: An FDA CBER Workshop for Patient Advocates
May 24, 2022
RegenMedEd Webinar: The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases
March 9, 2022
Frequently Asked Questions: Developing Potential Cellular and Gene Therapy Products (Final, August 20, 2026). This finalizes the November 2024 draft. It compiles OTP's answers to common sponsor questions across regulatory, CMC, pharmacology and toxicology, and clinical topics, and was created in response to a PDUFA VII commitment.
Potency Assessment of Active Immunotherapy Products (Draft, August 2026). The draft gives recommendations for developing potency assays for active immunotherapy products (ACTIMPs), including identifying potency-related critical quality attributes and selecting bioassays or physicochemical methods. It supplements the December 2023 draft Potency Assurance for Cellular and Gene Therapy Products. The deadline to submit comments to the docket is November 18.
Formal Meetings Between FDA and Sponsors or Applicants of PDUFA Products (Final, August 2026). The companion CBER SOPP 8101.1 took effect September 21.
Container Closure Systems for Human Drugs and Biological Products (Draft, August 2026).
CMC Flexibilities for CGT Products (Final, May 2026). CBER finalized guidance formalizing phase-appropriate CMC expectations for CGT products heading toward BLA. The guidance codifies reduced process validation requirements for Phase 1, clarifies comparability expectations for manufacturing changes during development, and supports a risk-based approach to CMC oversight.
Genome Editing Safety Assessments (Draft, April 2026). FDA issued draft guidance outlining safety assessment approaches for genome-edited gene therapy products, including expectations for off-target analysis.
Prior Knowledge / Plausible Mechanism Framework (Draft, June 2026). CBER issued draft guidance clarifying how sponsors may leverage existing nonclinical, clinical, and manufacturing knowledge to streamline development — reducing duplicative studies for individualized and platform-based CGT products.
Responding to FDA Form 483 Observations at the Conclusion of a Drug CGMP Inspection
E22 General Considerations for Patient Preference Studies
M4Q(R2) The Common Technical Document for the Registration of Pharmaceuticals for Human Use: Quality
Use of Bayesian Methodology in Clinical Trials of Drug and Biological Products
Accelerated Approval – Expedited Program for Serious Conditions
Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products Questions and Answers: Final 01/06/2025
Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Hepatitis B Virus (HBV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Hepatitis C Virus (HCV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Human Immunodeficiency Virus (HIV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Advanced Manufacturing Technologies Designation Program: Final 12/31/24
E11A Pediatric Extrapolation: Final 12/30/24
Purpose and Content of Use-Related Risk Analyses for Drugs, Biological Products, and Combination Products July 2024
Platform Technology Designation Program for Drug Development May 2024
Read Kim Benton's article regarding this guidance here.
Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry
Read Amanda Mack and Alicja Fiedorowicz's publication regarding this guidance here.
Considerations for the Use of Human-and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products; Draft Guidance for Industry April 2024
Read Blake Bergam and Sara Mills' article on this subject here.
Providing Regulatory Submissions in Electronic Format: IND Safety Reports Guidance for Industry April 2024
Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products March 2024
Early Alzheimer’s Disease: Developing Drugs for Treatment March 2024
Q14 Analytical Procedure Development Q2 March 2024
(R2) Validation of Analytical Procedures March 2024
Advanced Manufacturing Technologies Designation Program February 2024
Human Gene Therapy Products Incorporating Human Genome Editing January 2024
Potency Assurance for Cellular and Gene Therapy Products; Draft Guidance for Industry December 2023
Master Protocols for Drug and Biological Product Development December 2023
Rare Diseases: Considerations for the Development of Drugs and Biological Products December 2023
Data Standards for Drug and Biological Product Submissions Containing Real-World Data December 2023
Translation of Good Laboratory Practice Study Reports: Questions and Answer November 2023
Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory November 2023
Submitting Patient-Reported Outcome Data in Cancer Clinical Trials November 2023
Guidance for Industry: Voluntary Consensus Standards Recognitions Program for Regenerative Medicine Therapies October 2023
Guidance for Industry: Voluntary Consensus Standards Recognitions Program for Regenerative Medicine TherapiesData Standards for Drug and Biological Product Submissions Containing Real-World Data October 2023
Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities October 2023
Expedited IND Pilot (FDA, September 15, 2026). The pilot, part of HHS Operation TrialBlazer, pairs a sponsor with a qualified research institution and tests whether rolling review of IND components during the pre-IND phase reduces the risk of a clinical hold during the 30-day IND review. FDA expects to select 8 to 10 sponsor and institution pairs from applications due October 30, 2026, and states it retains full authority over clinical hold decisions.
Direct final rule on nonclinical testing terminology (September 21, 2026). The rule replaces "animal tests" with "nonclinical tests" across drug and biologics regulations to recognize methods such as human cell cultures and organs-on-chips, without removing animal study requirements or changing safety standards. A database of 25 new approach methodology examples was also launched.
Good Clinical Practice commentary from FDA center leaders (FDA Voices, September 3, 2026). Leaders of CDER, CBER, CDRH, and OCE wrote that GCP is "not optional," describing planned steps that include more foreign bioresearch monitoring for early-phase trials and notices to sponsors when a site cannot be inspected. Sponsors running first-in-human CGT studies ex-US should expect scrutiny of site data integrity.
CNPV program. The Casgevy pediatric approval (53 days from filing) is the eighth CNPV approval, reinforcing the program's short review clocks for selected products. No additional participants for the program have been announced since the departure of former Commissioner Marty Makary.
Operation TrialBlazer (HHS/FDA, June 22, 2026). HHS unveiled Operation TrialBlazer, a cross-agency initiative (FDA, NCI, NCATS, NIH, ARPA-H) aimed at keeping early-stage clinical research in the U.S. For CGT specifically, FDA is clarifying phase-appropriate IND expectations that the agency estimates could reduce time from drug identification to first-in-human trial by 6–12 months.
PreCheck Pilot Program (FDA, June 29, 2026). FDA selected seven companies for the PreCheck Pilot, a program to expedite domestic manufacturing facility approvals. Two of the selections note CGT manufacturing: Cellares Corp. (Bridgewater, NJ; cell-based gene therapies for oncology/hematology) and Kriya Therapeutics (Durham, NC; gene therapies for chronic diseases).
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry Submit comments by November 24, 2025
Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations; Draft Guidance for Industry Submit comments by November 24, 2025
Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry Submit comments by December 24, 2025
Recommendations to Reduce the Risk of Transmission of Mycobacterium tuberculosis (Mtb) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Guidance for Industry Final 01/06/2025.
Issued for immediate implementation but comments may be submitted.
Submit comments.
Considerations for Complying with 21 CFR 211.110
Draft 01/06/2025. Submit comments by 04/07/25
M15 General Principles for Model-Informed Drug Development:
Draft 12/30/24. Submit comments by 02/28/25
E6(R3) Good Clinical Practice: Annex 2:
Draft 12/30/24. Submit comments by 02/28/25
Protocol Deviations for Clinical Investigations of Drugs, Biological Products, and Devices:
Draft 12/30/24. Submit comments by 02/28/25
Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products; Draft Guidance for Industry:
Draft 11/19/24. Submit comments by 02/18/24
Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers:
Final 10/1/24. Allowing late comments.
Our first Regulatory Roundup LIVE was our Unbridled Excellence #4 and featured our unparalleled team of former FDA regulators discussing the latest and greatest regulatory news...that's DHC Master Principal Kimberly Benton and Master Practice Expert Don Fink, as well as Senior Principal Heath Coats, former Dark Horse Kevin Whittlese, and Senior Practice Expert Tal Salz. Go to the main Unbridled Excellence page to watch/download episode #4 on demand.