Current FCS Clinical Studies
FCS

 

VOLANESORSEN (The APPROACH Study)

(Currently in development by Akcea)

Apolipoprotein (ApoC-III) is a protein target for therapies that Akcea is developing to lower levels of triglycerides in the blood. High levels of ApoC-III slow down the metabolism and/or removal of triglycerides from the bloodstream, which can lead to high levels of triglycerides in the blood.8

Volanesorsen (IONIS-APOCIIIRx) is an antisense drug designed to reduce ApoC-III protein production and to lower triglycerides to treat patients with dyslipidemia.

Akcea completed a broad phase 2 program evaluating volanesorsen in patients with high, very high, and severely high triglycerides, in patients with high triglycerides and type 2 diabetes and in patients with FCS. In all patient groups studied, irrespective of their incoming triglyceride levels, reductions in in ApoC-III, triglycerides were observed.In addition, we observed significant improvements in glucose control with trends toward enhanced insulin sensitivity in volanesorsen-treated patients with type 2 diabetes.

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GLYBERA (Apilogene tiparvovec)

(Currently in development by uniQure)

Glybera (Alipogene tiparvovec) is a gene therapy that transfers a working copy of the LPL gene into cells via a carrier (vector).

Glybera is a gene therapy that is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells. In order to improve activity, uniQure uses a naturally occurring variant of the LPL gene that has higher enzyme activity than the normal version of the gene that encodes the protein. The Company produces Glybera using its insect cell-based manufacturing process. Clinicians administer Glybera in a one-time series of up to 60 intramuscular injections in the legs. The patient is administered spinal anesthesia or deep sedation during the procedure. In addition, an immunosuppressive regimen is recommended from three days prior to and for 12 weeks following Glybera administration.

Read More  (study ID NCTo2904772)