FDA Orders Halt to Trial of Novartis's Zolgensma

   By Carlo Martuscelli 
 

The U.S. Food and Drug Administration has ordered an ongoing clinical trial of Novartis's Zolgensma halted after adverse findings were detected in an animal study.

The stop concerns a clinical trial called Strong which is examining Zolgensma administered through an injection in the spinal column.

Novartis said Wednesday that the regulator asked it to pause the trial after the company reported nerve-cell inflammation, sometimes accompanied by cell degeneration, in animals.

The order has no impact on the already-approved, intravenous formulation of the gene therapy for spinal muscular atrophy, which continues to be available for patients in the U.S., the pharmaceutical company said.

The injection formulation of Zolgensma is being studied as a treatment for patients up to the age of five. The currently approved formulation can only be given to newborns and toddlers up to the age of two.

The company earlier this year discovered that data on mouse testing related to the manufacture of certain lots of Zolgensma had been manipulated by scientists. The FDA criticized Novartis for failing to disclose those concerns immediately, but said the manipulation didn't change its view that Zolgensma is safe and effective.

Despite the data manipulation scandal, sales of what is the world's most expensive drug were healthy in its first full quarter in the market, totaling around $160 million.

Write to Carlo Martuscelli at [e-mail]; @carlomartu

(END) Dow Jones Newswires

October 30, 2019 02:47 ET (06:47 GMT)

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