Pegaptanib

S01LA03

VEGF inhibitor

Template: Infobox chemical / molecular formula search available

Pegaptanib ( an acronym from the Engl. PEGylated aptamer inhibitor) is a VEGF inhibitor that is used as a drug for the treatment of wet age-related macular degeneration ( AMD).

Pegaptanib is a PEGylated, 2' -fluoro- pyrimidine and 2' -O -methyl- substituted purine 27-mer RNA aptamer ( NX1838 ) in December 2004 and was the first FDA-approved aptamer.

Development

Pegaptanib was developed by the company EyeTech Pharmaceuticals, Inc. and transferred by means of a license to Pfizer. Pfizer funded the late clinical trials and took over the marketing. Pfizer the active ingredient as in the vitreous body of the eye ( intravitreally ) administered injection solution is sold under the tradename Macugen ®.

Mechanism of Action

An aptamer is a single strand of nucleic acid that specifically binds to a specific target ( target molecule ) binds ( key - lock principle ). Pegaptanib binds specifically to VEGF - A165, an extracellular protein that plays an important role in the formation of new blood vessels (angiogenesis ) and for the increased permeability (permeability) plays of blood vessels. Both are pathologically important processes in the loss of vision by AMD.

By intravitreally, ie directly into the vitreous of the eye, injected aptamer to be stopped specifically neovascularization ( neovascularization ) by the responsible for this growth factor ( vascular endothelial growth factor ), VEGF is antagonized.

Effectiveness

Pegaptanib slows or stops the progression of moisture Makuladegneration. Compared with untreated patients, the incidence of severe visual loss is significantly reduced with long term use of pegaptanib. In a study published in 2004 was found by the use of pegaptanib compared with sham-treated controls ( placebo) significantly increased chance of stable or increasing visual acuity. However, the question is not clear whether other forms of therapy, such as the use of ranibizumab or PDT superior, or are equivalent and cheaper.

Dosage

Pegaptanib is injected in doses of 0.3 mg once in six weeks intravitreally.

Side effects

In most cases, side effects have been attributed to the injection method, see, therefore, with respect to the side effects associated with intravitreal injection