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2023-01-16|

EMA Plans to Issue Liver Failure Warning for Novartis’ Gene Therapy

by Reed Slater
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After Novartis reported two deaths in pediatric patients after receiving its spinal muscular atrophy (SMA) gene therapy, Zolgensma, the European Medicines Agency said it drafted a direct healthcare professional communication (DHPC) noting concerns about acute liver failure associated with the therapy.

Zolgensma’s Safety Concerns

Novartis’ Zolgensma is a gene therapy that uses an adeno-associated viral (AAV) vector that delivers a functional SMN gene to replace a missing or faulty SMN1 gene often found in SMA patients. In some cases, the therapy can elicit an immune response, activating capsid-specific T cells that may result in hepatotoxicity. 

In rare cases, like those reported in August last year, patients can develop acute liver failure. 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. 

The FDA approved Zolgensma in 2019 as the first gene therapy for children less than two years old with SMA. The U.S. label quickly adopted a black box warning for increased risks of acute liver failure. 

Related Article: Novartis Reported Two Deaths Following Zolgensma Gene Therapy

The EMA’s Proposed Dear Doctor Letter

During a Pharmacovigilance Risk Assessment Committee (PRAC) meeting, the EMA drafted a DHPC containing important safety information about Zolgensma. The committee did not publish the letter yet but plans to after forwarding the message to the committee for advanced therapies (CAT) and the human medicines committee (CHMP). 

The letter will include updated recommendations for monitoring liver function in patients who may receive Zolgensma and information about assessing liver damage after receiving the one-time gene therapy. It will also include details on responding to potential liver damage with corticosteroids. The EMA noted that if patients do not respond adequately to corticosteroids, physicians should consult a pediatric gastroenterologist or hepatologist expert to adjust the corticosteroid regimen.

Once individual committees within the EMA agree on the content of the DHPC, the agency will disseminate the message to healthcare professionals. Due to liver failure safety concerns, Novartis has distributed similar letters in the past in the U.S. regarding Zolgensma. 

With the acute liver failure safety concerns surrounding Novartis’ revolutionary SMA gene therapy, Zolgensma, the EMA will jump on board with a DHPC for healthcare professionals outlining guidelines and recommendations when administering the treatment. Despite its potential side effects, the drug remains the only approved gene therapy for the condition on the market, paving the way for future therapies. 

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