The first drug shown to significantly improve the vision of patients threatened by a major cause of blindness in the elderly won federal approval Friday.
The drug, called Lucentis, treats the wet form of age-related macular degeneration, a disorder where blood vessels behind the retina leak blood and fluid, worsening vision and often causing blindness. An estimated 90 percent of the 1.4 million Americans who have lost their eyesight due to the disorder have the wet form.
Lucentis, made by the South San Francisco, Calif., biotechnology company Genentech Inc., inhibits the growth of blood vessels when injected into the eye. Other Food and Drug Administration-approved treatments can arrest progression of the disease, which can lead to blindness in just weeks or months, but none has been shown to significantly reverse deteriorating vision.
Genentech may find Lucentis competing against another of its drugs, the cancer treatment Avastin. Avastin is increasingly used to treat macular degeneration for as little as $17 a dose. Lucentis is likely to cost upward of $2,000 a dose.
Lucentis and Avastin both block the same protein believed responsible for the blood vessel growth. Early results from Lucentis trials led doctors more than two years ago to begin experimenting with Avastin to treat age-related macular degeneration, commonly called AMD.
"It gave physicians and patients great hope we are going to be able to improve vision in our patients rather than just slow down the loss of vision," said Dr. George Williams, an American Academy of Ophthalmology spokesman, of the early results.
Since then, at least 10,000 macular degeneration patients have received Avastin injections, a so-called "off-label" use of the drug.
"It's become a worldwide phenomenon," said Dr. Philip Rosenfeld, a professor of ophthalmology at the Bascom Palmer Eye Institute in Miami who pioneered its use for AMD.
The Food and Drug Administration does not sanction using Avastin to treat macular degeneration. Genentech stresses that it has not studied the safety or effectiveness of Avastin in treating the disease, nor does it plan to do so.
"We believe Lucentis is a much better choice," said Dr. Hal Barron, Genentech's chief medical officer. "I really believe when treating patients, you have to look at the wealth of data supporting its use and the quality of that data."
The National Eye Institute, part of the National Institutes of Health, has received an outside proposal to conduct a study comparing Avastin and Lucentis in treating AMD, the government institute's Dr. Maryann Redford said.
Genentech engineered Lucentis to better penetrate the retina while reducing inflammation, Barron said. Lucentis also clears the body faster than Avastin, and better targets the protein important to vessel formation, he added.
Still, the anticipated cost of Lucentis may constrain its use, since Avastin is far cheaper when vials of the cancer drug are divvied up and used to treat macular degeneration.
"The bottom line is, wherever price does matter, Avastin will probably be used as first-line therapy. But wherever price is not an issue, like in Medicare patients and Medicaid patients, Lucentis will be used," Rosenfeld said.
Neither drug is a cure for macular degeneration; patients likely would have to receive injections five to seven times a year to ward off the eye problems associated with the disorder. The disease is named for the macula, the central portion of the retina responsible for detailed central vision.
The two other FDA-approved therapies to treat macular degeneration are Novartis AG/QLT Inc.'s Visudyne, which combines a light-sensitive drug and laser therapy to seal leaking blood vessels, and OSI Pharmaceuticals Inc./Pfizer Inc.'s Macugen, which is injected into the eye. Both slow the progression of the disorder but typically do not reverse its effects.
Lucentis' most commonly reported side effects include conjunctival hemorrhage, eye pain, floaters, increased eye pressure and inflammation.
In clinical trials, nearly 95 percent of participants given monthly injections maintained their vision at one year, compared to the approximately 60 percent who did after receiving another treatment, the FDA said. Approximately one-third of the Lucentis patients also reported improved vision after a year.
Genentech said it would start shipping Lucentis Friday. It didn't immediately disclose the price.