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:  Gene-editing advances put us at the dawn of a revolution in medicine

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

ADVERTISEMENT

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 Daily Digest
Biotech Facts & Fallacies
GLP Podcasts
Infographic: Here’s where GM crops are grown around the world today

Infographic: Here’s where GM crops are grown around the world today

Do you know where biotech crops are grown in the world? This updated ISAAA infographics show where biotech crops were ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

* indicates required
Email Lists
Send this to a friend