Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion - Summary of Product Characteristics

What this platform has drawn from it — 4 claims

• The UK licence for etranacogene dezaparvovec, a gene therapy delivered to liver cells, contraindicates it in patients with known advanced hepatic fibrosis or cirrhosis, and in patients with active acute or uncontrolled chronic infections. Section 4.3 Contraindications, second and third bullets
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• Before etranacogene dezaparvovec is given, the licence requires that the patient's liver transaminases are assessed and that liver ultrasound and elastography are performed, along with laboratory tests to evaluate for active hepatitis B and C. Section 4.4 Special warnings and precautions for use, baseline assessment
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• After etranacogene dezaparvovec is given, the licence directs that transaminases are closely monitored, for example once per week for at least three months, and that a corticosteroid taper is considered if alanine aminotransferase rises above the upper limit of normal or to double the patient's baseline. Section 4.4, subheading 'Hepatotoxicity'
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• Because of the risk of malignancy from vector integration, the licence directs that patients who have received etranacogene dezaparvovec undergo regular liver ultrasound screening and are monitored for alpha-fetoprotein elevations, for example annually, for at least five years after administration. Section 4.4, subheading 'Risk of malignancy as a result of vector integration'
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