Skip to main content
Premium Trial:

Request an Annual Quote

Pause Button Hit

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. 

The Scan

Genetic Ancestry of South America's Indigenous Mapuche Traced

Researchers in Current Biology analyzed genome-wide data from more than five dozen Mapuche individuals to better understand their genetic history.

Study Finds Variants Linked to Diverticular Disease, Presents Polygenic Score

A new study in Cell Genomics reports on more than 150 genetic variants associated with risk of diverticular disease.

Mild, Severe Psoriasis Marked by Different Molecular Features, Spatial Transcriptomic Analysis Finds

A spatial transcriptomics paper in Science Immunology finds differences in cell and signaling pathway activity between mild and severe psoriasis.

ChatGPT Does As Well As Humans Answering Genetics Questions, Study Finds

Researchers in the European Journal of Human Genetics had ChatGPT answer genetics-related questions, finding it was about 68 percent accurate, but sometimes gave different answers to the same question.