CGTLive's 2024 Pillars of Progress: Most-Read FDA News in Cell and Gene Therapy

For all of 2024, our team was following along the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

The team spent all year bringing the latest information to the website's front page, from major data publications and presentations to FDA decisions and major medical meetings. Agency decisions were among the key portions of our coverage, with our team covering submissions, approvals, delays, and other actions across the breadth of medical specialties in search of the biggest news in cell and gene therapy.

Here, we'll highlight some of the most-read content from CGTLive's FDA coverage this year. Click the buttons to read further into these stories.

FDA Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic Leukemia

“Adult ALL is an extremely aggressive cancer, and there is a high unmet medical need that exists in the treatment of patients with this disease once they relapse, where historically they suffer from poor outcomes. This milestone approval, based on the demonstrated clinical benefit of Aucatzyl, brings new hope for adult patients with r/r B-ALL.”
—Elias Jabbour, MD

In November, the FDA announced that approved obecabtagene autoleucel (Aucatzyl; Autolus Inc), known colloquially as obe-cel, a CD19-directed genetically modified autologous T-cell immunotherapy, for the treatment of adults with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL). The therapy was approved based on the findings of the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed rates of overall complete remission (CR) above 60% and a median duration of remission beyond 12 months for those who achieved complete remission within 3 months.²

FDA Approves Sarepta’s DMD Gene Therapy Elevidys for Expanded Indication

"Today’s expansion of the Elevidys label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle. The initial approval of Elevidys was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence, and the improvements in the trajectory of the disease we have seen to date across studies."
—Jerry Mendell, MD

In June, the FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), for an expanded indication in the disease. The therapy is now approved for ambulatory patients (via traditional approval) and nonambulatory patients (via accelerated approval) with a confirmed mutation in the DMD gene who are 4 years of age or older and who do not have any deletion in exon 8 or exon 9 in the gene.

FDA Approves PTC Therapeutics’ Gene Therapy Kebilidi for AADC Deficiency

"PTC has once again pioneered a new approach to treating highly morbid neurologic diseases. I am proud of our team's unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States."
—Matthew B. Klein, MD

Also in November, PTC Therapeutics’ eladocagene exuparvovec, a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, was approved by the FDA for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. Notably, the indication covers the full spectrum of disease severity and the approval constitutes the first gene therapy for direct administration to the brain to be approved in the United States. The product will be marketed in the US under the name Kebilidi; in the European Union and United Kingdom, it is marketed as Upstaza.

FDA Approves Mesoblast’s Remestemcel-L for Steroid-Refractory GvHD

“Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children. This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies.”
—Peter Marks, MD, PhD

Just this month, in December the FDA approved Mesoblast’s allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy remestemcel-L for the treatment of steroid refractory acute graft versus host disease (GvHD) in children aged 2 months or older. Notably, the product is the first MSC therapy to have been approved by the FDA. It will be marketed under the name Ryoncil.

FDA Finalizes Sets of Guidelines for Both CAR-T Products and Genome Editing Products

"We first posted the draft guidance on considerations for the development of CAR T-cell products in March of 2022... [and] we received over 650 comments from approximately 85 stakeholders, and we really appreciate your thoughtful insights into where more clarity would be helpful. After consideration of each comment and with revisions as needed, the final guidance was then published at the beginning of 2024."
—Kim Schultz, PhD

After finalizing the guidance in January, in March, the FDA's Center for Biologics Evaluation and Research (CBER) hosted a webinar highlighting key points from the final version of the CAR-T guidance document on March 7, 2024. In the webinar, a recording of which is now available on the FDA's website, the speakers covered the main points from the general, CMC, nonclinical, and clinical considerations for CAR-T development from the document. The webinar also included a question-and-answer segment (which begins about 30 minutes into the presentation).

Click here to watch the FDA's webinar on the CAR T guidance.