Products
Alipogentiparvovec has been approved in the EU as a solution for injection since 2012 (Glybera). It was the first gene therapy to be released in Europe. The drug’s high price is controversial.
Structure and properties
The drug contains a highly active variant of the lipoprotein lipase gene (S447X) in a viral vector. This consists of:
- Protein envelope from adeno-associated virus serotype 1 (AAV1).
- Cytomegalovirus promoter
- Woodchuck hepatitis virus post-transcriptional regulatory element.
- AAV2-derived terminal inverted repetitive sequences (inverted terminal repeats).
Effects
Alipogentiparvovec (ATC C10AX10) contains the gene for lipoprotein lipase, which is produced in muscle after administration. The virus cannot replicate in the body and the gene is not integrated into human DNA but is located extrachromosomally.
Indications
For the treatment of familial lipoprotein lipase deficiency (LPLD) with pancreatitis flares.
Dosage
According to the SmPC. The drug is injected intramuscularly into the muscles of the legs once several times.
Contraindications
For complete precautions, see the drug label.
Adverse effects
The most common possible adverse effects include leg pain, headache, fatigue, and increased body temperature.
