AveXis, manufacturer of a drug recently approved to treat young spinal muscular atrophy (SMA) patients, has announced a partial hold on Zolgensma that is administered via spinal canal injection.
The hold was placed by the U.S. Food & Drug Administration (FDA) after AveXis found dorsal root ganglia (DRG) mononuclear cell inflammation in 12 animals who were given Zolgensma via intrathecal administration in a pre-clinical study.
In an Oct. 31 announcement, AveXis said the inflammation was “sometimes accompanied by neuronal cell body degeneration or loss. DRG inflammation can be associated with sensory effects such as tingling, prickling, chilling, burning, or numb sensation. The DRG inflammation seen in this pre-clinical animal study was not seen in prior animal studies with AVXS-101,” also known as Zolgensma.
AveXis said the FDA hold does not apply to Zolgensma that is administered intravenously.
“We recognize this news may be disappointing and cause concern among the community,” AveXis said in its announcement. “We want to reinforce that we remain confident the overall benefit-risk profile for patients on treatment is favorable. We will work diligently with FDA to identify any additional actions necessary to resume dosing in the AVXS-101 IT clinical trials.”
Zolgensma is currently FDA approved only for SMA patients under 2 years of age. AveXis’s work is focusing on making the drug available to older SMA patients, as well.
It’s been a rough few months for AveXis and its parent company, Novartis.
In May, the FDA approved Zolgensma to treat children who have SMA and are under age 2. But in August, the FDA said AveXis employees had “manipulated” Zolgensma study data, and that the company was aware of the manipulation before the FDA approved the drug.
AveXis, however, didn’t alert the FDA of the data manipulation until after the FDA had already approved the drug.
According to a report from industry publication FiercePharma, Novartis learned of the results of the animal intrathecal studies in March, and had planned to tell the FDA about those findings in a September update, but failed to include the information in its report.
Upon realizing the mistake, Novartis notified the FDA in October. The manufacturer then announced the FDA’s partial hold on Oct. 30.
Zolgensma is a one-dose genetic treatment for SMA. Earlier this year, it made mainstream headlines and was called the country’s most expensive drug due to its $2.1 million-per-dose price tag.
