EMA safety committee seeks to warn doctors about Zolgensma risks

Jan. 18, 2023

In response to two fatal cases of acute liver failure in patients treated with Novartis' Zolgensma, the EMA's safety group — the Pharmacovigilance Risk Assessment Committee (PRAC) — wants to issue an alert to health care professional containing additional information on the gene therapy.

The warning, known as direct healthcare professional communication (DHPC), would inform health care professionals of the fatal cases of liver failure and the updated recommendations for monitoring liver function, assessing suspected liver injury after infusion and further advice regarding tapering the corticosteroid treatment.

Back in August, Novartis reported two patient fatalities due to acute liver failure following treatment with Zolgensma gene therapy. The drugmaker notified health authorities, including the FDA, in markets where the drug was being sold. The two fatal cases of acute liver failure took place in Russia and Kazakhstan after 5 to 6 weeks of Zolgensma infusion and about 1-10 days following the initiation of corticosteroid taper, Novartis reported.

Zolgensma was approved by the FDA in May 2019 for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy. In the U.S., it carries a 'black box' warning for acute serious liver injury and acute liver failure.

A year after its U.S. approval, the therapy was granted conditional approval in the EU by the EMA. Now, the DHPC for Zolgensma will be forwarded to EMA’s committee for advanced therapies and to EMA’s human medicines committee (CHMP). Once adopted, Novartis will be responsible for disseminating the new guidance to health care professionals in the EU.