Thu, Apr 11, 2019 - 5:50 AMTHE Food and Drug Administration on Tuesday approved an osteoporosis drug that represents the first new treatment approach in nearly two decades - a strategy based on a rare gene mutation in people with bones so dense that they never break.
"This is an extraordinarily important drug," said Dr Richard Bockman, chief of the endocrine service at the Hospital for Special Surgery in New York."It's a true bone-building drug that takes advantage of the underlying biology of bone." In one study, spinal fractures occurred in 127 of 2,046 patients taking the new drug, compared with 243 of 2,047 taking aldendronate, an older drug.
The new drug has a striking backstory. In 1964, researchers began studying an unusual group of Afrikaner patients in South Africa. They were tall and heavy, but not fat. Instead, their bones were large and dense. In osteoporosis, the balance is disrupted - more bone is broken down than is made. Once bone density increased, patients could stop taking the sclerostin-blocking drug and switch to an older drug to maintain the new bone. Animal studies were successful, as were clinical trials, culminating in two large studies involving more than 10,000 postmenopausal women.