Novartis Trial of World's Most Expensive Drug Halted Over Safety Concerns
October 30 2019 - 9:04AM
Dow Jones News
By Denise Roland
Novartis AG has paused a clinical trial of its Zolgensma gene
therapy after a separate study on animals raised concerns about
side effects, dealing a setback to the company's efforts to broaden
the market for the world's most expensive drug.
The trial treated children up to five years old with spinal
muscular atrophy, or SMA, an inherited muscle-wasting disease,
through a spinal injection of the $2.1 million per patient
drug.
The U.S. Food and Drug Administration ordered a partial hold on
the trial after Novartis reported that primates receiving Zolgensma
through spinal injection suffered nerve cell inflammation,
sometimes accompanied by cell degeneration.
The halt doesn't affect the use of the therapy in children up to
the age of two, which was approved earlier this year, because that
version is given through intravenous injection rather than through
the spine. Zolgensma provides a working version of the gene at
fault in SMA.
The news is a setback for Novartis, which hoped the trial
targeting older children with type 2 SMA -- a milder form of the
disease -- would pave the way to treat a much broader group of
patients.
There are around 4,800 older children living with type 2 SMA in
the U.S. Only around 300 of those would be eligible to receive
Zolgensma in its already-approved form.
Jefferies analyst Peter Welford said in a note to clients that
losing that potential market would erode Zolgensma's sales
potential by nearly a third. He forecasts peak sales at about $2.8
billion but says that would fall to $2 billion if the company is
unable to sell the treatment for older children with type 2
SMA.
Mr. Welford and other analysts believe the stop is likely to
delay, rather than derail, the trial. Novartis said no children who
had received the spinal injection of Zolgensma had suffered nerve
inflammation. It also said no other animal studies had shown this
side effect. Earlier this month, Novartis reported the trial had so
far shown positive results. To resume the trial, Novartis will have
to provide data to the FDA to reassure the agency there are no
safety concerns over the spinal injection in children.
Despite its eye-catching price tag, Novartis said Zolgensma has
sold well since its launch in late May, overcoming concerns about
whether insurers would cover the treatment as well as a
data-manipulation scandal at the unit that makes it.
Novartis defended the price saying it would cost half that of
the current standard treatment, Biogen Inc.'s Spinraza, over a
10-year period. Spinraza, which is a continuing treatment, costs
$750,000 for the first year and $375,000 for each year after
that.
The company also pointed to a study by the independent nonprofit
Institute for Clinical and Economic Review, which said Zolgensma is
worth $2.1 million when given to newborns who haven't yet developed
symptoms of the disease.
The drug halts, rather than reverses, the condition -- so the
earlier a patient receives treatment, the better the outcome.
Novartis hopes that as newborn screening gains traction, the
majority of patients will fall into this category.
Novartis also faced scrutiny after it discovered earlier this
year that testing data related to the manufacture of certain
Zolgensma lots had been manipulated by scientists at the unit that
makes it. The FDA criticized Novartis for failing to disclose those
concerns immediately but said the manipulation didn't change its
view that the drug is safe and effective.
Write to Denise Roland at Denise.Roland@wsj.com
(END) Dow Jones Newswires
October 30, 2019 08:49 ET (12:49 GMT)
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