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EMA Pharmacovigilance Risk Assessment Committee Advice on Safety-Related Aspects of the Gene Therapy

Long-term follow-up due to risk of cancer
19 Feb 2021

As part of its advice on safety-related aspects to other European Medicines Agency (EMA) committees, the Pharmacovigilance Risk Assessment Committee (PRAC) discussed at its meeting held from 8 to 11 February 2021 direct healthcare professional communications (DHPCs) containing important safety information for Strimvelis, Venclyxto and Zolgensma. 

Strimvelis: Long-term follow-up due to risk of cancer 

The DHPC is intended to warn doctors of the risk that the use of the gene therapy Strimvelis could lead to genetic mutations with the potential to cause cancer in patients who receive this medicine. Strimvelis is a medicine to treat severe combined immunodeficiency due to adenosine deaminase deficiency. This is a rare inherited condition in which there is a change in the gene needed to make an enzyme adenosine deaminase that is crucial in maintaining healthy white blood cells. Doctors are advised to monitor patients long-term for cancerous changes with at least annual visits for the first eleven years and then at 13- and 15-years post treatment with Strimvelis. 

PRAC has recommended this DHPC in the context of a careful analysis of a single case of acute leukaemia (lymphoid T cell leukaemia) reported in a patient who was treated with Strimvelis in 2016, almost 5 years prior to the cancer diagnosis. 

Venclyxto: New measures for minimising the risk of tumour lysis syndrome 

The DHPC intends to inform healthcare professionals on new recommendations and measures for the mitigation of the risk of tumour lysis syndrome in patients treated with venetoclax (Venclyxto), and the related updates to the summary of product characteristics. 

Zolgensma: Recommendations to manage risk of thrombotic microangiopathy 

The DHPC is intended to warn doctors of the risk of thrombotic microangiopathy (an acute and life-threatening condition characterised by thrombocytopenia, haemolytic anaemia and acute kidney injury) following administration of onasemnogene abeparvovec (Zolgensma), a gene therapy for patients with spinal muscular atrophy. The DHPC enhances awareness of this risk and advises on the need for prompt clinical management. 

The DHPCs for the gene therapies Strimvelis and Zolgensma will be sent to the EMA’s Committee for Advanced Therapies and then to EMA’s Committee for Medicinal Products for Human Use (CHMP) for a final opinion. The DHPC for Venclyxto will go directly to the CHMP. Following the CHMP decision, the DHPCs will be disseminated to healthcare professionals by the marketing authorisation holder, according to agreed communication plans, and published on EMA’s website and in National registers in EU Member States.

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