Cell and Gene Therapy Decisions Due Soon

In the next six months, the FDA expects to issue decisions on three novel therapies: Orca-T, Deramiocel (CAP-1002), and Anito-cel (anitocabtagene autoleucel). Each targets a different illness, utilizing uniquely engineered cells to maximize efficacy while minimizing serious side effects.

(1)   Orca-T

  • PDUFA Date: 7/6/2026

  • Indications: Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, and Myelodysplastic Syndromes

  • Description: A high-precision, allogeneic T-cell therapy immunotherapy. It is manufactured from matching healthy donors and consists of  individually formulated (1) hematopoietic stem and progenitor cells, (2) high-purity Tregs, and (3) conventional T cells (Tcons).

  • Clinical Significance: For patients with hematological malignancies, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only potentially curative therapeutic option. However, Graft-vs-Host Disease (GvHD) remains a serious complication impacting recovery and is one of the primary causes of mortality. In its pivotal Phase 3 trial, Orca-T significantly improved survival free from moderate-to-severe chronic GvHD (78% compared to just 38% with conventional therapy) while also drastically reducing rates of infection and non-relapse mortality.

  • References

    • Everett H. Meyer, Amandeep Salhotra, Arpita P. Gandhi, Jeremy Pantin, Sagar S. Patel, Rasmus T. Hoeg, Alexandra Gomez-Arteaga, Rawan Faramand, Roni Tamari, Edmund K. Waller, Satyajit Kosuri, Antonio M. Jimenez Jimenez, Jennifer Holter-Chakrabarty, Bhagirathbhai Dholaria, Yi-Bin Chen, Betty K. Hamilton, John Magenau, Alireza Eghtedar, Joshua M. Murray, Anna Pavlova, Nathaniel B. Fernhoff, James Scott McClellan, M. Scott Killian, Ai Li, Robert S. Negrin, Caspian Oliai; Orca-T vs allogeneic hematopoietic stem cell transplantation (Precision-T): a multicenter, randomized phase 3 trial. Blood 2026; 147 (11): 1168–1177. doi: https://doi.org/10.1182/blood.2025031313

    • Orca Bio Announces FDA Review Extension of BLA for Orca-T for the Treatment of Hematologic Malignancies. News release. Orca Bio. April 1, 2026. Accessed June 24, 2026. https://orcabio.com/orca-bio-announces-fda-review-extension-of-bla-for-orca-t-for-the-treatment-of-hematologic-malignancies/

(2)   Deramiocel (CAP-1002)

  • PDUFA Date: 8/22/2026

  • Indications: Duchenne Muscular Dystrophy Cardiomyopathy

  • Description: Human allogeneic cell therapy containing cardiosphere-derived cells (CDCs) derived from heart tissue Rather than directly replacing muscle tissue, CDCs act by secreting vesicles called exosomes, which modify immune responses and provide potent anti-fibrotic (scar-reducing) benefits.

  • Clinical Significance: DMD causes progressive muscle degeneration affecting both skeletal and heart muscles. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. In the pivotal Phase 3 HOPE-3 trial, Deramiocel achieved a statistically significant slowing of skeletal muscle disease progression (by nearly 54% compared to placebo) and 91% slowing in decline of cardiac function measured with left ventricular ejection.

  • References

(3)   Anito-cel (anitocabtagene autoleucel)

  • PDUFA Date: 12/23/2026

  • Indications: Relapsed or Refractory Multiple Myeloma (RRMM)

  • Description: An autologous CAR T-cell therapy that targets the B-cell maturation antigen (BCMA). It is built with a novel, compact synthetic binding protein known as Arcellx's "D-Domain". This combination may allow for the removal of multiple myeloma cells without severe immunotoxicity.

  • Clinical Significance: Multiple myeloma is a difficult-to-treat blood cancer, and heavily pre-treated patients often see standard therapies fail. Anito-Cel achieves highly efficient binding and optimal tumor-killing while rapidly detaching from the target, which is intended to reduce toxicity and cell exhaustion. In the Phase 2 registrational iMMagine-1 trial, the therapy yielded an exceptional ~97% overall response rate with 74% achieving a stringent complete response or complete response per International Myeloma Working Group (IMWG) criteria. The progression-free survival (PFS) rates were 82.1% at 12 months, 67.4% at 18 months and 61.7% at 24 months. It is currently in Phase 3 trials (iMMagine-3 trial).

  • References

Article by Kathy Clark, RN, BSN, CMCN, RIT, Vice President, Director of Managed Care. For more information about how the impact on your plan, please contact your Summit ReSources care specialist.