Animal safety concerns prompt FDA to halt study testing Zolgensma gene therapy for spinal muscular atrophy

wnmktkpturbxy iowjkytblndu n exmjlknzhjnwnmnjawmtdjzmvimi qcgeslqmaom hcm d jmfzqmgzqhc
Zolgensma. Image: Novartis

The Food and Drug Administration has halted a clinical trial of Novartis’ Zolgensma gene therapy due to a safety concern found in an animal study, the company said [October 30].

The hold affects the Novartis (NVS) clinical trial known as STRONG, which was testing a higher dose of Zolgensma administered by spinal injection to older children with spinal muscular atrophy (SMA). It does not affect the already approved treatment of infants and children.

Novartis said its subsidiary AveXis informed regulators about findings from an animal study that showed “dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.” The clinical significance of this adverse safety signal is not known, but it can be associated with “sensory effects,” the company added.

Related article:  Viewpoint: Why you should give your DNA to NIH’s ‘All of Us’ initiative

Halting the STRONG clinical trial is a setback for Novartis’ effort to expand the use of Zolgensma to older patients with SMA.

Novartis said it has seen no reports of “sensory effects” in patients and is working with the FDA to resolve safety concerns and resume dosing of Zolgensma in the clinical trial.

Read full, original post: Novartis’ Zolgensma gene therapy study halted by FDA on animal safety concerns

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Video: Test everyone – Slovakia goes its own way to control COVID

Video: Test everyone – Slovakia goes its own way to control COVID

As Europe sees record coronavirus cases and deaths, Slovakia is testing its entire adult population. WSJ's Drew Hinshaw explains how ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend