FDA Orders Halt to 'Strong' Trial of Novartis's Zolgensma -- Update
October 30 2019 - 5:55AM
Dow Jones News
--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 carlo.martuscelli@dowjones.com;
@carlomartu
(END) Dow Jones Newswires
October 30, 2019 05:40 ET (09:40 GMT)
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