STAT+: Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments have hit the market.
Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of genetic medicine, allowing some children who may have once died in infancy to grow up. But there was a catch: It was only available for children under age 2.
The limitation was logical. The pivotal trial enrolled only babies under 6 months. Two-year-olds born with the most common and severe form of SMA would likely have lost too many neurons to benefit from the therapy. Yet there were many older children and even some adults with debilitating but more mild disease who might be treated.
Those patients waited years. A safety issue in animals led the FDA to put a 21-month hold on a trial testing an alternative administration of Zolgensma, given directly into the spinal cord for more direct access to neurons, in older children.
