Novartis'
Zolgensma study halted by FDA amid safety questions
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[October 30, 2019]
By John Miller
ZURICH (Reuters) - U.S. regulators have
halted a trial of Novartis's Zolgensma treatment after an animal study
raised safety concerns, the company said on Wednesday, in a setback for
the drugmaker's plan to expand its use to older patients.
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The U.S. Food and Drug Administration's partial hold on the
so-called STRONG trial impacts patients aged up to five with spinal
muscular atrophy (SMA) who were to receive a higher dose of the gene
therapy via a spinal infusion.
The hold was issued after Novartis told health authorities about the
animal study's findings that showed dorsal root ganglia (DRG)
mononuclear cell inflammation, a neurological condition sometimes
accompanied by nerve damage or loss.
Novartis shares fell 1% at 0715 GMT, while other European drugmakers
rose. Analysts said more information was needed to assess whether
these findings could have an impact on Zolgensma's long-term
prospects.
"We need to better understand, what the issues are and whether there
really is a read-through to the human trial," Vontobel analyst
Stefan Schneider said.
Novartis said it was working with regulators, in hopes of having the
hold released.
Zolgensma, whose $2.1 million list price makes it the world's
most-expensive treatment, is already approved for children aged up
to two with the deadly muscle wasting disease and is given by
infusion into the young patients' veins.
The medicine had $160 million in sales in its first full quarter on
the market.
The STRONG study is meant to underpin its expanded approval for
older children, where Novartis would also compete with Biogen's
Spinraza that is already on the market, and Roche's investigational
risdiplam that is due to seek approval this year.
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Roche shares were up 0.7%.
"The clinical significance of the DRG inflammation observed in this
pre-clinical animal study is not known and was not seen in prior
animal studies," Novartis said in a statement.
"We are working with health authorities to confirm further guidance
to clinical investigators."
The partial FDA hold halts enrollment of patients getting a higher
dose of Zolgensma, also known as AVXS-101, via an spinal infusion,
also called intrathecal administration.
Low- and mid-dose portions of the STRONG trial have already been
completed, as has presentation of interim results.
"Of note, we have completed a thorough review of human safety data
from all available sources to date and no adverse effects related to
sensory changes have been seen," Novartis said.
(Editing by Deepa Babington)
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