FDA Orders Halt to 'Strong' Trial of Novartis's Zolgensma -- Update

--FDA has halted a clinical trial of a version of gene-therapy Zolgensma

--The drug remains on the market in its current formulation

--Novartis said it will work with the health regulator to decide how to continue to study

 
   By Carlo Martuscelli 
 

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

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

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 Wednesday.

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

The Swiss drug maker said it is unsure of the clinical impact of the inflammations detected in the animal study, as they hadn't been previously observed. A review of the human safety data from the drug doesn't show any adverse effects that would be associated with inflammation, Novartis said.

The company said it is still confident in the treatment, and that it would speak with health authorities to determine how to continue the study. Meanwhile, ongoing intravenous Zolgensma trials will continue.

The injection formulation of Zolgensma is being studied as a treatment for patients up to the age of five. The currently-approved version of the drug 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 05:40 ET (09:40 GMT)

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