Zolgensma, as posted earlier here, is the promising life-saving one-time gene therapy for patients with spinal muscular atrophy (SMA). Shocking news about patient death after Zolgensma was published this week.

Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma. As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported. “While this is important safety information, it is not a new safety signal and we firmly believe in the overall favorable risk/benefit profile of Zolgensma,” Novartis said in a statement emailed to news organizations.

Novartis did not reveal information about the patients, who were both children. However, the company did disclose that one of the fatal cases of acute liver failure took place in Russia and the other, in Kazakhstan. Both cases occurred at approximately five to six weeks post Zolgensma infusion, and approximately 1–10 days following the initiation of corticosteroid taper, Novartis stated.

Implications of patient death after Zolgensma

Zolgensma label already stated that the product can increase liver enzyme levels and cause acute serious liver injury or acute liver failure. Patients are to receive an oral corticosteroid before and after infusion with Zolgensma and undergo regular blood tests to monitor liver function. Now Novartis is extending the warning to specify that fatal acute liver failure has been reported.

These deaths are expected to renew the public discussion over the safety of adeno-associated virus (AAV) gene therapies such as Zolgensma. They are to open broader debates on safety and management of systemic AAV therapies in fragile or very young patients.

Zolgensma remains a promising life-saving gene therapy. It replaces the function of the missing or nonworking SMN1 gene. Without enough SMN protein, motor neuron cells stop working and muscles become weak. With each day that passes without treatment, motor neuron cells become weak and eventually lose all function and die. As a result, things like breathing, eating, swallowing, and speaking become difficult to impossible. Once these cells stop working, the condition is likely to become life threatening.

Source: Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma. Genetic Engineering and Biotechnology News. 12 August 2022.

See also: SMA Gene Therapy.

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