Cell therapy weekly: US FDA approval of a spinal muscular atrophy gene replacement therapy
This week: the US Food and Drug Administration (FDA; MD, USA) has approved a gene replacement therapy for spinal muscular atrophy (SMA) following encouraging clinical data and a consistent safety profile, and the European Commission has approved Breyanzi® for use in patients with relapsed or refractory mantle cell lymphoma (r/r MCL). Plus, a new partnership has been formed to streamline cell and gene therapy development in the UK.
The news highlights:
- FDA approves gene replacement therapy for spinal muscular atrophy
- European Commission approves Breyanzi in r/r MCL
- Partnership aiming to streamline cell and gene therapy in the UK
FDA approves gene replacement therapy for spinal muscular atrophy
The FDA has approved Novartis’ (Basel, Switzerland) gene replacement therapy, Itvisma® (onasemnogene abeparvovec-brve), for children aged two years and older, teens, and adults with SMA caused by a mutation in the SMN1 gene. SMA is a rare genetic disease that leads to progressive muscle weakness due to insufficient production of SMN protein, essential for motor neuron function. Individuals with more copies of the SMN2 gene, which produces limited SMN protein, often experience milder forms of SMA.
Following positive results from the registrational Phase III STEER and open-label Phase IIIB STRENGTH studies, the FDA has expanded the approval of Itvisma, making it the first and only gene replacement therapy for this broad population of patients. Patients showed significant improvements in motor function and sustained effects over 52 weeks of follow-up. The therapy demonstrated a consistent safety profile, with common adverse events including upper respiratory infections, pyrexia and vomiting.
“The FDA’s approval of intrathecal onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups,” said John W Day, Director of the Division of Neuromuscular Medicine at Stanford University School of Medicine (CA, USA). “This achievement is not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”
European Commission approves Breyanzi in r/r MCL
Following pivotal Phase I clinical trial results, the European Commission has approved Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed CAR T-cell therapy developed by Bristol Myers Squibb (NY, USA), for adult patients with r/r MCL after at least two prior systemic therapies, including a Bruton tyrosine kinase inhibitor.
Breyanzi demonstrated strong efficacy in pivotal trials, achieving an overall response rate of 82.7% and a complete response rate of 71.6%. Responses were rapid, with a median time to onset of 0.95 months, and durable, with 41.2% of patients still in response at 24 months.
The safety profile was consistent with prior Breyanzi studies, with the majority of adverse events, including cytokine release syndrome and neurologic toxicities, occurring within 14 days post-infusion. This approval is applicable to all European Union members states, as well as the European Economic Area countries (Iceland, Norway and Liechtenstein).
“This approval for Breyanzi in [r/r MCL] marks another important step as we continue to deliver on the promise of cell therapy for more eligible patients across Europe – the fourth approval for Breyanzi in Europe,” said Emma Charles, senior vice president, Europe Region, Bristol Myers Squibb. “While frontline therapies have advanced over the years for this rare but aggressive form of non-Hodgkin lymphoma, the vast majority of patients relapse or become resistant and face reduced survival outlook, leaving a critical need for new treatment options. Breyanzi has the opportunity to address a treatment gap for this patient population based on its demonstrated clinical benefit.”
Partnership aiming to streamline cell and gene therapy in the UK
eXmoor Pharma (Bristol, UK) and the Royal Free London NHS Foundation Trust’s Cell and Vector Innovation Centre (CVIC; London UK) have partnered to streamline cell and gene therapy development in the UK. By combining CVIC’s early-phase clinical manufacturing expertise with eXmoor’s later-phase clinical manufacturing capabilities, the collaboration aims to simplify processes, accelerate timelines and reinforce the UK’s position as a leader in advanced therapeutics.
The partnership will focus on aligning technology transfer, quality systems, regulatory processes, and training programs to create a seamless pathway from proof of concept to commercial launch, aiming to support academics, clinical and commercial innovators.
“For the UK’s cell and gene therapy sector to truly thrive, key learnings must flow freely across institutional and commercial boundaries,” said Owen Bain, Director of CVIC, Royal Free London NHS Foundation Trust, said. “This collaboration is designed to do exactly that – connecting NHS early-phase experience with eXmoor’s development and commercial manufacturing expertise. By sharing practical know-how, aligning standards, and building mutual understanding, we can catalyze the manufacturing pathway for the next wave of innovators and create a stronger, more resilient UK ecosystem for advanced therapies.”
