Robert M Califf, MD, MACC, on 2023’s Landmark FDA Approvals of Cell and Gene Therapies

“If you love biological science, or you're concerned about the well-being of people that currently have untreatable diseases or diseases for which the [current] treatments are not great, this is as exciting as it can get.”

2023 was a major year for cell therapy and gene therapy, especially when it comes to regulatory decisions in the United States. Notably, the FDA approved 2 gene therapies for sickle cell disease (SCD), a gene therapy for dystrophic epidermolysis bullosa, a cell therapy for type 1 diabetes, and a gene therapy for severe hemophilia A.^1-5 In addition to these, many more investigational cell therapies and gene therapies are currently in clinical development for therapeutic areas as wide-ranging as cardiology, endocrinology, dermatology, lysosomal disorders, oncology, hematology, neurology, ophthalmology, and autoimmune diseases. As such, the field of cell therapy and gene therapy is only expected to grow in importance in the coming years as more and more of these investigational treatments are put in front of the FDA.

As part of a larger look back at the FDA’s decisions in 2023, CGTLive™’s sister publication HCPLive™ sat down with Robert M Califf, MD, MACC, the commissioner of food and drugs at the FDA, to discuss the progress made in gene therapy and cell therapy this year. Califf spoke about the importance of past research efforts, especially The Human Genome Project, in bringing the field to where it is today. Califf also higlighted the recent approvals of both Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia) for SCD, both which were announced on December 8, 2023. He also expressed optimism for the future, but pointed out the need to continue to pay close attention to safety and to work with the Centers for Medicare & Medicaid Services (CMS) regarding economic affordability for these emerging treatment modalities.

REFERENCES
1. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 7, 2023. Accessed December 19, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
2. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed December 19, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
3. Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech. May 19, 2023. Accessed December 19, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene
4. FDA approves first cellular therapy to treat patients with type 1 diabetes. News release. FDA. June 28, 2023. Accessed December 19, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes
5. U.S. Food and Drug Administration approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe hemophilia A. News release. BioMarin Pharmaceutical Inc. June 29, 2023. Accessed December 19, 2023. https://investors.biomarin.com/2023-06-29-U-S-Food-and-Drug-Administration-Approves-BioMarins-ROCTAVIAN-TM-valoctocogene-roxaparvovec-rvox-,-the-First-and-Only-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A