New Macular Degeneration Drug on the Horizon

New Macular Degeneration Drug on the Horizon

A company, Ophthotech, recently announced their drug is now funded to enter Phase II FDA trials for testing.

Their new drug, E10030, is an aptamer (similar to an antibody) targeted against “Platelet Derived Growth Factor.”  The aptamer is designed to specifically bind to PDGF and prevent “plugging-in” to its receptor.

The drug is novel for two reasons; 1) it is directed at a second protein PDGF and not VEGF (vascular endothelial growth factor) and 2) it is being tested in combination with anti-VEGF medication Lucentis.

The company feels that the combination of the two drugs gives a better improvement than Lucentis/anti-VEGF alone.

What Does This Mean? Phase II FDA trials  are meant to test for safety, but more importantly, are also designed to test for efficacy of a drug.  In other words, Phase II trials determine how well a drug works.  Many drugs fail at this point.

The identification of another factor leading to wet macular degeneration is intriguing; however, as are the claims that the two agents work better than anti-VEGF alone.

Also, I bring this drug to your attention to highlight that it is a potential new drug and not even close to approval, yet you may hear that this is a new “treatment.”  It is not and has a long way to go.  Many people will be touting this as a new therapy.

The FDA has a total of 4 clinical trials.  We are several years away from significant results.


Randall V. Wong, M.D.
Ophthalmologist, Retina Specialist
Fairfax Virginia

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