In a setback for Novartis' efforts to expand the use of its gene therapy drug Zolgensma to older patients, the FDA halted the drugmaker's clinical trial after it found a safety concern in an animal study, according to STAT.
The STRONG trial was testing a higher dose of Zolgensma administered by a spinal injection to older children with spinal muscular atrophy. Pausing the trial doesn't affect the dosage already approved to treat infants and children.
AveXis, a subsidiary of Novartis, reportedly told regulators it found "dorsal root ganglia mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss" in the animal study. The clinical significance of the condition isn't known, but can cause sensory effects, according to STAT.
Novartis told STAT it has seen no evidence of sensory effects in patients and is working with the FDA to resolve safety concerns and resume the clinical trial.
The setback follows a scandal in which the drugmaker admitted it knew preclinical data was falsified before the drug was approved but didn't tell the FDA until after its drug received approval.
Zolgensma is the world's most expensive drug, with a price tag of $2.1 million for the one-time treatment. Earlier this month, the drugmaker reported it used the drug to treat 100 patients since it received approval in May and brought in $160 million in the third quarter, higher than analysts' expectations.
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