"... Some initial tests of the drug have been completed and were successful. A
trial of fifteen patients has found that at 24 months, all of the
babies that were treated were alive and did not need permanent
ventilation. Without treatment, ninety percent of SMA1 patients must be
permanently ventilated by that age. In addition, eleven of twelve babies
that received the recommended dosage were able to sit unassisted for
over five seconds, which is “a milestone never achieved in the natural
history of SMA Type 1,” according to Novartis."
"..The Food and Drug Administration (FDA) had previously designated
Zolgensma as a Breakthrough Therapy and has now granted it Priority
Review. Novartis is expecting regulatory decisions from the FDA this
May.
"This important step by the FDA brings us ever closer to delivering
Zolgensma to patients with SMA Type 1. Babies affected by this rare
disease are currently faced with debilitating disease progression and
lifelong invasive chronic treatment. As a one-time infusion that
addresses the genetic root cause of SMA without the need for repeat
dosing, Zolgensma represents a potentially significant therapeutic
advance for these patients and their families," said AveXis president
David Lennon.
"The introduction of one-time, potentially curative therapies will
require rethinking how our healthcare system manages diagnosis,
treatment, care and associated costs for patients with genetic disease,”
he added. “Novartis and AveXis are proud to lead the way toward a
modern healthcare system built on the tremendous value of truly
innovative and transformative medicines that could bend the curve of
life. We are committed to flexibly partnering with healthcare
stakeholders to ensure appropriate access to our medicines."