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 Novartis also said on Thursday that babies with muscle-wasting
spinal muscular atrophy (SMA) treated before symptoms emerge were
meeting normal development milestones, a welcome announcement amid
revelations of data manipulation that have shrouded Zolgensma.
Novartis has been investigating the death of a 6-month-old patient
in a European trial. Novartis executives now say investigators and
the coroner concluded the immediate cause was brain damage from
oxygen deprivation after respiratory distress, not brain damage from
a toxic drug agent.
"There was no evidence of any inflammatory central nervous system
process or toxic or drug-related brain damage," said Olga Santiago,
chief medical officer of Novartis's gene therapy unit AveXis, on a
call with reporters.
While gene therapy may have contributed to some of the reasons why
the baby was hospitalized in London, such as abnormal liver function
and low blood pressure, Novartis said those are side effects it has
long known about.
"We had been concerned about the potential... that Zolgensma might
have been causing brain inflammation," said AveXis Chief Executive
Dave Lennon. "In this case, the original diagnosis, and that
association, hasn't held true."
Zolgensma, the world's most expensive one-time medical treatment,
has provided hope for families facing a devastating diagnosis of SMA,
the leading genetic cause of infant death.
It has also made negative headlines: in August, the U.S. Food and
Drug Administration began probing manipulation of early data by
AveXis scientists, saying it could result in criminal or civil
action.
While Novartis replaced AveXis employees and has since promised to
speed disclosures of problematic data, the FDA left Zolgensma on the
market after concluding it is safe and effective.
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Study data released by Novartis on Thursday at the European
Pediatric Neurology Society Congress in Greece appear to underscore
the FDA's decision, particularly for babies tested for SMA at birth
and who receive treatment before showing floppy muscles and other
symptoms.
Among 10 patients measured so far in Novartis's SPRINT study, none
was delayed in unsupported sitting. Six could sit at an average of
7.6 months, while three stood with assistance at an average 10.1
months.
Without treatment, they faced death or permanent breathing help.
EARLY DIAGNOSIS
Like a previous study in kids treated before symptoms emerge with
the first approved SMA treatment, Biogen's $750,000 Spinraza,
Novartis's results underscore newborn screening's importance in
helping children achieve the best outcomes, Santiago said. [https://reut.rs/2kmuH6H]
Kids treated with Spinraza or Zolgensma after SMA symptoms emerge
often survive, but may never stand and face a lifetime of costly
medical challenges. While U.S. states are adding newborn SMA
screening, Novartis said Europe is lagging.
"It's critical to diagnose early and to begin treatment as early as
possible to stop the irreversible motor neuron loss, and make the
achievement of motor milestones such as crawling, sitting and
walking possible," Santiago said.
(Reporting by John Miller; Editing by Michael Shields)
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