A drug developed by a Duke University researcher to treat severe gout was recently approved by the U.S. Food and Drug Administration, capping a 17-year journey from idea to market.
The drug, which will be sold under the name Krystexxa, has been hailed as a lifesaver by many of the patients who tested it. Gout, a painful buildup of a waste product called uric acid, can be debilitating for those who cannot take traditional therapies.
"This is good stuff," said L.T. Matthews, 77, of Burlington, who received the drug in clinical trials at Duke. He said his gout flare-ups three years ago were so painful he was often bedridden. After a regimen of the drug, which is administered in an intravenous drip twice a month, he was pain-free.
Dr. Michael Hershfield, a rheumatologist at Duke and the Durham VA Hospital, began work on the drug in the early 1990s after treating many patients who, like Matthews, were completely wiped out by the condition.
"There's an estimated 3 (million) to 5 million patients with gout in the United States, and more than 90 percent of them do well on available medications," Hershfield said. "But there's a subset of them ... who have progressive gout, and who cannot take or respond to treatment, and they progress to terrible disease."
A doctor and biochemist, Hershfield was uniquely positioned to tackle the problem. He had recently scored a major success helping bring to market another drug that was aimed at a rare immune disorder in babies.
That drug fused a large molecule called a polymer to an enzyme the babies lacked. The polymer wasn't absorbed by the body, so the enzyme could circulate much longer to provide its healing power. The technology of fusing the so-called PEG polymer with biological therapies has since created other long-lasting treatments.
Hershfield was interested in using that approach for a gout therapy, but he faced numerous obstacles. Money was tight, the interest of corporate partners waned, and experiments that showed promise in animals had flaws when tested in humans.
But Hershfield and his research partner at Duke, Susan Kelly, kept working, convinced that the drug would help desperate gout patients. Finally in 2008, trial results showed the drug was effective in reducing uric acid levels in a substantial number of patients.
The drug, which is manufactured by New Jersey-based Savient Pharmaceuticals, was on its way to winning FDA approval in 2009 — only to be tripped again. Federal authorities raised concerns that the manufacturing process had been altered and demanded corrections.
Another year passed, and the decision on the drug's fate was set for Tuesday.
"We were on pins and needles," Hershfield said.
After watching the clock all day, Hershfield headed home. Just as he was pulling out of the parking garage at work, his wife called with the news.
"Truly it was more of a relief," he said, noting that a rejection would have killed any further exploration of the therapy. "I couldn't imagine the drug not being available to patients."
Matthews, the Burlington patient, said he, too, couldn't bear the idea of the drug failing.
"It was a lifesaver for me," he said. "I was really at the point I didn't want to continue living. I was that bad off."