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On November 8, 2024, the U.S. FDA issued a historic approval of the seventh CAR-T cell therapy to be approved in the U.S. and the 11th CAR-T therapy to be approved globally. This approval was for Autolus Inc.’s Aucatzyl (obecabtagene autoleucel), a CD19-directed genetically modified autologous T cell immunotherapy, for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Autolus is a U.K. biotech company that develops novel gene-modified T cell therapies for the treatment of cancer.
To date, the other U.S. FDA approved therapies have been:
- Kymriah (tisagenlecleucel)
- Yescarta (axicabtagene ciloleucel)
- Tecartus (brexucabtagene autoleucel)
- Breyanzi (lisocabtagene maraleucel)
- Abecma (idecabtagene vicleucel)
- Carvykti (ciltacabtagene autoleucel)
Most of the FDA approved CAR-T cell therapies have been approved in numerous healthcare markets worldwide.
Within China, two CAR-T therapies have also been approved by the National Medical Products Administration (NMPA), Relma-cel by JW Therapeutics and Yuanruida by CASI Pharmaceuticals (in partnership with Juventas Cell Therapy). Within India, two CAR-T cell therapies have also received approval from the Central Drugs Standard Control Organization (CDSCO), NexCAR19 by ImmunoAct and Qartemi by Immuneel Therapeutics.
Efficacy and Safety
Efficacy of Autolus’s Aucatzyl was assessed in a recent human trial, FELIX (NCT04404660). This was an open-label, multicenter, single-arm trial that enrolled adult patients with relapsed or refractory CD19-positive B-cell ALL. Enrolled patients were required to have relapsed following a remission lasting 12 months or less, relapsed or refractory ALL following two or more prior lines of systemic therapy, or disease that was relapsed or refractory 3 or more months after allogeneic stem cell transplantation.
The major efficacy outcome measures were rate and duration of complete remission (CR) achieved within 3 months after infusion. Additional outcome measures were rate and duration of overall complete remission which includes complete remission and complete remission with incomplete hematologic recovery (CRi), at any time. Of the 65 patients evaluable for efficacy, 27 patients (42%; 95% confidence interval [CI]: 29%, 54%) achieved CR within 3 months. The median duration of CR achieved within 3 months was 14.1 months (95% CI: 6.1, not reached).
The prescribing information has a boxed warning for cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and T cell malignancies.
This application was granted regenerative medicine advanced therapy (RMAT) designation and orphan drug designation.
U.S. FDA Approval of Aucatzyl
The FDA approval of Aucatzyl is historic because it introduces a novel CAR-T cell therapy specifically for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)—a highly aggressive cancer with limited treatment options. Aucatzyl’s personalized, targeted approach offers new hope to patients who have not responded to conventional treatments, potentially extending remission periods and improving outcomes.
In particular, Aucatzyl may cause fewer side effects than Gilead’s Tecartus in patients with leukemia. Aucatzyl is a competitor to Tecartus by Gilead because both are CAR-T cell therapies approved to treat aggressive blood cancers, specifically targeting CD19 on B-cells, and both aim to address relapsed or refractory cases where traditional treatments have failed.
With similar mechanisms of action but different proprietary technologies, Aucatzyl and Tecartus will compete to offer patients more effective, personalized options in the growing CAR-T therapy market. Given this reality, it will be interesting to see Gilead’s strategic response and whether it will be collaborative (such as an acquisition) or competitive (for example, increased marketing spend).
This approval also spotlights the massive momentum underway within the global CAR-T field, with Autolus emerging as the latest competitor within this lucrative race. For more coverage of this landmark approval, view the U.S. FDA announcement.
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