Macugen (pegaptanib)

The following drug information is obtained from various newswires, published medical journal articles, and medical conference presentations.

Approval Status:

Approved December 2004

Specific Treatments:

Macular Degeneration

Therapeutic Areas

General Information

Macugen (pegaptanib) is a selective vascular endothelial growth factor (VEGF) antagonist.

Macugen is indicated for the treatment of neovascular (wet) age-related macular degeneration. According to the present product label, Macugen (0.3 mg) should be administered once every six weeks by intravitreous injection into the eye to be treated.

There are 15 million people in the United States living with some form of AMD, with more than 1.6 million experiencing the active blood vessel growth and blood vessel leakage associated with neovascular AMD.

Clinical Results

FDA approval of Macugen was based on results from two controlled, double-masked, randomized trials (study EOP1003 & EOP1004) enrolling a total of 1,200 patients with neovascular AMD. Subjects received 0.3 mg, 1 mg or 3 mg if Macugen by injection or a control (sham) treatment every 64 days for 48 weeks. The median age of the patients was 77 years. All subjects enrolled had classic, occult, or mixed lesions of up to 12 disc areas and baseline visual acuity in the study eye between 20/40 and 20/320.

Results showed that treatment with Macugen demonstrated statistically significant less vision loss compared to sham treatment. Results showed that among patients receiving 0.3 mg of Macugen, 70% lost less than three lines of vision on the eye chart, compared with 55% of patients receiving control treatment (P<.0001). The results demonstrated a 27% relative treatment effect for Macugen treated patients compared to controls with respect to three line loss. Macugen also helped limit progression to legal blindness, by 50 % compared to controls, in the study eye.More sham treated patients (75/296) received PDT than Macugen 0.3 mg treated patients (58/294).

On average, Macugen 0.3 mg treated patients and sham treated patients continued to experience vision loss. The rate of vision decline in the Macugen treated group was slower than the rate in the patients who received sham treatment. In both studies, the primary endpoint was the proportion of patients losing less than 15 letters of visual acuity, from baseline up to 54 week assessment.

Side Effects

Adverse events associated with the use of Macugen may include (but are not limited to) the following:

  • Ocular Discomfort
  • Eye Pain
  • Endophthalmitis
  • Reduced Visual Acuity
  • Visual Disturbance
  • Corneal Edema
  • Blurred Vision
  • Dizziness

Mechanism of Action

Pegaptanib is a selective vascular endothelial growth factor (VEGF) antagonist. VEGF is a secreted protein that selectively binds and activates its receptors located primarily on the surface of vascular endothelial cells. VEGF induces angiogenesis, and increases vascular permeability and inflammation, all of which are thought to contribute to the progression of the neovascular (wet) form of age-related macular degeneration (AMD), a leading cause of blindness.

Literature References

Bayes M, Rabasseda X, Prous JR. Gateways to clinical trials. Methods Find Exp Clin Pharmacol. 2002 Nov;24(9):615-43.

Gragoudas ES, Adamis AP, Cunningham ET Jr, Feinsod M, Guyer DR; Pegaptanib for neovascular age-related macular degeneration. N Engl J Med. 2004 Dec 30; 351(27):2805-16.

Vinores SA. Technology evaluation: pegaptanib, Eyetech/Pfizer. Curr Opin Mol Ther. 2003 Dec; 5(6):673-9.

Additional Information

For additional information regarding Macugen or macular degeneration, please visit The Macugen Web Site