As 2025 comes to a close, the FDA has approved a novel gene therapy and expanded indications for two cell therapies as described below. Looking into 2026, prospective therapies on the near horizon include a gene therapy for Fabry’s disease as well as CAR-T therapies for Stiff Man Syndrome, light chain amyloidosis, and CD5-positive hematologic malignancies. Following is the current list of FDA approved cell and gene therapies.
On December 8, 2025, the FDA approved Omisirge® (omidubicel-onlv) for an expanded indication for the treatment of adults and pediatric patients 6 years and older with severe aplastic anemia (SAA) following reduced intensity conditioning. Omisirge initially received FDA approval in 2023 to treat adults and patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen.
List Price: $512,070
https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-severe-aplastic-anemia
Full Label: Omisirge-final-PI.pdf
On December 9, 2025, the FDA approved Waskyra® (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). Waskyra is an autologous hematopoietic stem cell-based gene therapy indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate, and no suitable HLA-matched related stem cell donor is available.
List Price: Not disclosed yet.
Full Label: Package Insert - WASKYRA
On December 4, 2025, the FDA granted approval to Breyanzi® (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T-cell therapy for adult patients with marginal zone lymphoma (MZL) who have failed treatment with or relapsed after two or more prior lines of therapy.
Full Label: Package Insert - Breyanzi
Article by Kathy Clark, RN, BSN, CMCN, RIT, Vice President, Director of Managed Care. For more information about how this may affect your plan, please contact your Summit ReSources care specialist.