Cell therapy weekly: FDA approves CAR-T cell therapy for relapsed/refractory B-cell acute lymphoblastic leukemia
This week: Terumo Blood and Cell Technologies (Terumo BCT; CO, USA) has launched a new business unit to improve patient care throughout the treatment journey. Plus, the US Food and Drug Administration (FDA; MD, USA) has approved a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency and a CAR-T cell therapy for relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).
The news highlights:
- Enhancing patient access to advanced therapies
- FDA approval of AADC deficiency gene therapy
- FDA approves CAR-T cell therapy for r/r B-ALL
Enhancing patient access to advanced therapies
Terumo BCT has introduced its Global Therapy Innovations business unit, focused on expanding patient access to advanced therapies and enhancing care standards throughout the treatment journey. This new unit is structured to provide integrated expertise in disease management, utilizing the company’s extensive involvement at various stages of patient care.
“We are now ideally situated as a singular partner, with a holistic view to make connections among cell quality, process standardization and cell collection access, as well as to understand the impact of these factors further downstream in the cell therapy manufacturing process,” said Veerle d’Haenens, General Manager at Global Therapy Innovations. “This will enable us to bring together a variety of stakeholders to help unlock patient access to therapies.”
FDA approval of AADC deficiency gene therapy
The FDA has approved KEBILIDI™ (eladocagene exuparvovec-tneq), a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy developed by PTC Therapeutics (NJ, USA) for treating AADC deficiency. This marks the first gene therapy approved in the US that is directly administered to the brain.
AADC deficiency is a rare, life-threatening genetic disorder that impairs dopamine production. KEBILIDI aims to deliver a functional DDC gene directly into the putamen, thereby increasing the AADC enzyme to restore dopamine synthesis. The therapy received accelerated approval based on safety and efficacy results from an ongoing global clinical trial, which demonstrated de novo dopamine production and improvements in motor development milestones.
“PTC has once again pioneered a new approach to treating highly morbid neurologic diseases,” stated Matthew Klein, CEO of PTC Therapeutics. “I am proud of our team’s unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States.”
FDA approves CAR-T cell therapy for r/r B-ALL
The FDA has granted marketing approval for Autolus Therapeutics’ (Stevenage, UK) CAR-T cell therapy, AUCATZYL®, for the treatment of r/r B-ALL in adults. This approval, which did not require a risk evaluation mitigation strategy, was based on the FELIX clinical trial, where the therapy demonstrated encouraging remission rates and manageable side effects.
“We are so pleased to now be able to offer AUCATZYL, our first commercial product, to adult r/r B-ALL patients in the US. This approval would not have been possible without the support of all the patients, their families and caregivers, their treating physicians and the nurses and investigators at the treatment centers – thank you,” expressed Christian Itin, CEO of Autolus. “This milestone is the culmination of many years of hard work, the foundational work by our partners at [University College London (UK)] and the unwavering commitment of our internal team, our external partners and shareholders. This is a proud day for Autolus.”
