2023 may have been a breakthrough year for cell and gene therapies with the FDA approving a record of seven novel treatments, but 2024 did not fall behind in the number of greenlit therapies. With seven new approvals and three expanded indications, the cell and gene therapies authorized by the agency last year cover a variety of therapeutic areas, including beta thalassemia and solid tumor cancer.
The list of therapies to reach the finish line in 2024 included a number of firsts. Iovance Biotherapeutics’s Amtagvi was the first one-time T cell therapy for solid tumor cancer and the first treatment option for adults with advanced melanoma after anti-PD-1 and targeted therapy. Lenmeldy from Orchard Therapeutics for children with early-onset metachromatic leukodystrophy brought a treatment option to patients who previously had none. Autolus Therapeutics achieved its first-ever commercial approval with Aucatzyl while PTC Therapeutics’ Kebilidi became the one and only gene therapy directly administered to the brain.
Vertex Pharmaceuticals/ CRISPR Therapeutics’ co-developed Casgevy, which in 2023 became the first medicine using CRISPR/Cas9, a novel gene-editing technology, for sickle cell disease, was approved last year for the treatment of transfusion-dependent beta thalassemia.
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Accelerated Approvals
What the future holds for cell and gene therapy approvals within the FDA remains to be seen. The former US FDA top official for vaccines and cell and gene therapies Peter Marks supported the agency’s accelerated approval pathway to expedite the approval of these therapies for life-threatening conditions. However, since Mark’s departure and the turmoil that has set upon the agency, it is still unclear how the accelerated approvals will evolve.
