FDA approves treatment for growth disease acromegaly
Wednesday, March 26th 2003, 12:00 am
News On 6
WASHINGTON (AP) _ Sufferers of a rare disease that causes out-of-control growth are about to get a novel treatment that targets one of the condition's key villains.
The Food and Drug Administration approved Somavert on Wednesday as a treatment for acromegaly. The injected drug blocks the effects of excess growth hormone.
An estimated 40,000 people worldwide have acromegaly, which can make them abnormally tall, cause bone deformities and swell their facial features, hands and feet. Severe joint pain is common, and patients are at high risk of death from heart disease, diabetes and cancer spurred by the condition's excess hormones.
The disease killed the wrestler and actor Andre the Giant, best known for playing Fezzik in the 1986 movie, ``The Princess Bride''. He died in 1993, aged 46.
Acromegaly occurs when a non-cancerous tumor on the pituitary gland causes secretion of excessive growth hormone, which in turn causes dangerous overproduction of a second hormone called IGF-1.
Removing the pituitary tumor is the first step. But often growth hormone levels don't return to normal for years, so patients also need radiation or drug therapy to suppress growth hormone _ but some aren't helped.
Pharmacia Corp.'s Somavert doesn't suppress growth hormone but prevents its bad action, by blocking special cell receptors needed to trigger excess IGF-1, explained FDA's Dr. David Orloff.
More than 90 percent of patients who had failed other treatments saw their IGF-1 levels return to normal with daily Somavert injections, he said.
Normalizing IGF-1 is expected to lessen, if not prevent, acromegaly's worst symptoms. Orloff called Somavert an ``extremely promising'' treatment.
Pharmacia Corp. refused to reveal how much it will charge for Somavert, even though sales are set to begin next month. But treatments for rare diseases can cost tens of thousands of dollars a year.
Side effects include injection-site irritation, sweating, headache and fatigue.
Patients also must undergo regular liver testing during the first six months of Somavert use, the FDA warned. During studies, two patients experienced very high levels of liver enzymes that signal organ damage.
Somavert, known chemically as pegvisomant, is the first drug to target growth-hormone receptors, and scientists are beginning to study whether that specialized action might help other ailments, too, such as diabetes-caused blindness and nerve damage.