EMA Seeks to Issue Guidance on Liver Damage From Novartis' Gene Therapy

By Reuters Staff

January 13, 2023

(Reuters) -The European Medicines Agency (EMA) said on Friday it plans to issue advice for physicians using Novartis AG's Zolgensma, calling on them to monitor patients for any liver injury after treatment.

The statement follows two deaths due to liver failure after treatment with the gene therapy against spinal muscular atrophy, reported by Novartis in August.

Spinal muscular atrophy is a group of rare 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.

Novartis said it had notified health authorities of the deaths and sent letters to update healthcare providers on liver safety in markets including the United States in August.

In Europe, however, such letters had been the subject of discussions with EMA, but Novartis would soon be cleared to send them in EU member states, it added.

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.

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.

(Reporting by Raghav Mahobe, Manas Mishra in Bengaluru and Ludwig Burger in Frankfurt; Editing by Shounak Dasgupta, Shailesh Kuber, Kirsten Donovan)

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