The hold places a pause on enrollment in the high-dose cohort of the company's Phase I study of intrathecal AVXS-101, a different administration for the marketed therapy Zolgensma. It does not affect the marketed product and is unrelated to an early data manipulation scandal.
The company posted strong sales for Zolgensma, which the FDA approved five months ago for treating infants with spinal muscular atrophy. Strong and growing sales were also reported for the breast cancer drug Piqray and the CAR-T cell therapy Kymriah.
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Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2.
The Aug. 23 document from AveXis, posted on the FDA website Tuesday, fingered "two AveXis senior executives" who had been terminated. Brothers Brian and Allan Kaspar were ousted from AveXis last month, though the former denied any wrongdoing.
Novartis subsidiary AveXis is presenting Phase III data at a European pediatric neurology conference on the therapy, Zolgensma, which won approval in May for spinal muscular atrophy in infants.
Subsidiary AveXis said the two executives being replaced had not been involved in operations since May and were no longer with the company. Another publication reported that they had been ousted in connection the scandal.
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Five Democratic senators - two running for president - wrote in a letter to the FDA's acting commissioner that the scandal highlighted industry "privilege and greed."
In a statement Tuesday, an FDA official wrote that subsidiary AveXis disclosed manipulation of mouse data for the gene therapy Zolgensma, which the agency approved in May for spinal muscular atrophy. The official wrote that civil or criminal penalties were possible.
The gene therapy will be priced at $2.125 million, or $425,000 per year for five years, under an installment-based payment program for which Novartis is partnered with specialty pharmacy Accredo.