EU spotlight on Novartis gene therapy after patient deaths
The agency will urge them to monitor patients for any liver injury after treatment.
The announcement by the EMA last Friday follows reports of two fatal cases of acute liver failure after the administration of Zolgensma in August.
The two known liver failure cases occurred in pediatric patients in Russia and Kazakhstan. Both patients received corticosteroids in hopes of reversing the liver damage.
Spinal muscular atrophy (SMA) is a group of rare genetic disorders which affect the nerve cells and cause muscle wasting and weakness.
Novartis gained Zolgensma as part of its $8.7bn acquisition of AveXis Inc in 2018; gaining US Food and Drug Administration (FDA) approval the following year. Known as the world’s most expensive drug, the one-dose treatment costs $2m.
Sales for Zolgensma reached $1.4bn in 2021, up 47% on the year before. Steady US sales were joined by expanded access in Europe and other markets, with the drug getting the EU nod in 2020.
“Healthcare professionals should promptly assess patients with worsening liver function tests and/or signs or symptoms of acute liver illness,” said the agency, following a discussion at the meeting of its Pharmacovigilance Risk Assessment Committee (PRAC).
And the EMA said that if patients do not respond adequately to treatment with corticosteroids, treating physicians should consult specialists and consider adjusting the corticosteroid regimen.
The advice for healthcare professionals has yet to be approved by further expert panels at EMA, however, and that green light can typically take a few weeks, noted Reuters.
