Onasemnogen-Abeparvovec

Products

Onasemnogene bebeparvovec was approved in the United States in 2019 as a suspension for intravenous infusion (Zolgensma).

Structure and properties

It is a gene therapy using the -gene, in which adeno-associated serotype 9 (AAV9) viruses are used as vectors. The gene is contained in the form of double-stranded DNA in the capsid of the nonenveloped virus. The virus is not capable of replication.

Effects

The goal of treatment is to introduce the human SMN gene into cell nuclei so that sufficient functional SMN protein can be produced. The DNA is not integrated into the host cell DNA. It is present in the cell nuclei as a stable so-called episome. Onasemnogen abeparvovec crosses the bloodbrain barrier and has a rapid onset of action.

Indications

For the treatment of children younger than 2 years with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene.

Dosage

According to the SmPC. The drug is administered as an intravenous infusion and as a single dose. The dose depends on body weight.

Contraindications

For complete precautions, see the drug label.

Adverse effects

The most common potential adverse effects include increased aminotransferase levels and vomiting. Severe liver disease has been reported. Appropriate precautions must be observed.