EMA seeks to issue guidance on liver damage from Novartis’s gene therapy

The European Medicines Agency (EMA) said recently it is looking into issuing advice for physicians using Novartis AG’s Zolgensma, calling on them to monitor patients for any liver injury after treatment.

The statement follows recent deaths due to liver failure after treatment with the gene therapy, the EMA said.

Novartis in August reported two patient fatalities due to acute liver failure following treatment with Zolgensma, which is used to treat spinal muscular atrophy.

Spinal muscular atrophy is a group of genetic disorders which affect the nerve cells and cause muscle wasting and weakness.

The advice for healthcare professionals has yet to be approved by further expert panels at EMA, which typically takes a few weeks.

A Novartis spokesperson did not have an immediate comment, when contacted by Reuters.

EMA said that if patients do not respond adequately to treatment with corticosteroids, treating physicians should consult specialists and consider adjusting the corticosteroid regimen.

Infusion drug Zolgensma competes with Roche’s oral drug Evrysdi and Biogen’s spinal injection Spinraza.

It won conditional EU approval during early 2020 and it costs more than $2 million per patient.

Edits by EP News Bureau