Novartis has paused a trial of its Zolgensma gene therapy that was seeking to expand the drug's market, the Wall Street Journal reports.
The US Food and Drug Administration approved Zolgensma in May to treat children under the age of two years with spinal muscular atrophy. In a new trial, Novartis was hoping to show that Zolgensma — which has a hefty list price of $2.1 million — could treat children up to the age of five years, according to the Journal.
But Novartis reported to the FDA that primates that received Zolgensma as a spinal injection — how the drug was given to the older patients; the younger patients receive it intravenously — experienced nerve cell inflammation and, in some cases, cell degeneration, it adds. This lead the agency to order a partial hold on the trial, the Journal says.
It reports that some analysts think this will be a temporary delay, as no children in the study had experienced that effect and as the firm announced earlier this month that initial results appeared promising.
The development of Zolgensma has also been a source of scandal, as some early testing data of the drug was manipulated, leading Novartis to replace two top executives at its AveXis unit.