Gene and Cell Therapy Update

November ushered in two new therapies, AUCATZYL® (obecabtagene autoleucel), a CAR-T therapy which was approved on 11/8/2024, and Kebilidi™ (eladocagene exuparvovec-tneq), a gene therapy which was approved on 11/13/2024.

AUCATZYL is a CD19-directed genetically modified autologous T-cell immunotherapy indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The total recommended dose of obecabtagene autoleucel is 410 X 106 CD19 chimeric antigen receptor (CAR)-positive viable T cells to be administered as a split dose infusion on Day 1 and Day 10 (±2 days) based on bone marrow blast assessment and preceded by fludarabine and cyclophosphamide lymphodepleting chemotherapy. The list price is $525K and does not include lymphodepleting chemotherapy, hospitalization, or administration fees.

Kebilidi™ [eladocagene exuparvovec-tneq, PTC-AADC (Upstaza in the UK)], is a gene therapy for the treatment of children and adult patients with aromatic L–amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. This is the first-ever FDA approval for gene therapy directly administered to the brain. Administration of KEBILIDI occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. The administration procedure is performed by a qualified neurosurgeon in centers specialized in stereotactic neurosurgery.

Use in Specific Populations:

  • Pediatric Use: The safety and efficacy of KEBILIDI have not been studied in pediatric patients younger than 16 months.

  • Geriatric Use: Clinical studies of KEBILIDI did not include patients 65 years of age and older.

The price for this drug in the UK is $3.8M and does not include the administration and facility costs; the US price has not yet been disclosed.

Article by Kathy Clark, RN, BSN, CMCN, Vice President, Director of Managed Care. For more information about how this may affect your plan, please contact your Summit ReSources care specialist. The following sources were used as reference material for this article: