FDA advisers recommend approval of growth hormone

WASHINGTON -- Injecting growth hormone into very short but otherwise healthy children won grudging approval from a government advisory panel Tuesday, amid concern about the cost and trouble it would take to increase their adult height by roughly 2 inches.

Growth hormone has been used for 16 years to treat children who are extremely short because their bodies don't naturally produce the substance, and to treat a handful of other growth-stunting diseases. Some 200,000 children worldwide have taken it.

Now one manufacturer, Eli Lilly & Co., is seeking Food and Drug Administration approval to formally market its brand of growth hormone, Humatrope, for children who don't have those medical conditions.

The FDA's scientific advisers decided that the company's studies clearly showed children grew between 1.5 inches and 2.8 inches taller after about four years of growth-hormone shots than they otherwise would have.

Lilly would restrict the growth hormone to the abnormally short -- boys predicted to be shorter than 5-feet, 3-inches as adults, and girls shorter than 4-feet, 11 inches, Lilly stressed.

Lilly counts some 400,000 such children ages 7 to 15, but predicts that only about 10 percent ultimately would receive growth hormone because of tight restrictions it plans on eligibility, and because many families simply won't want to endure six shots a week for years.

Cosmetic use questioned

The FDA has long fought cosmetic use of growth hormone, and now is struggling to define just what is meaningful, medically appropriate use of the drug -- without opening the floodgates to normal children just yearning for a few extra inches.

"Is there a need for growth-enhancing therapy" in abnormally short but otherwise healthy children? asked FDA endocrinology chief Dr. David Orloff.

The panel of scientific advisers agonized over the decision, with many questioning if such small improvement helped children's quality of life enough to justify spending $10,000 to $25,000 a year for the drug -- and getting so many shots.

"I'm worried about the medicalization of shortness," said women's health specialist Nancy Worcester of the University of Wisconsin-Madison, the panel's consumer representative.

"We are talking about treating otherwise perfectly normal kids who are short for five to 10 years," with little information about long-term side effects, complained panelist Dr. Deborah Grady of the University of California, San Francisco.

Lilly plans tight restrictions on the drug's availability. Instead of selling it regularly through pharmacies, it can only be prescribed by certain specialists and will be shipped by specially appointed drug stores to patients who undergo a battery of growth tests.

Panelists couldn't say the proper age at which to begin treatment, or how to tell which child should stop the shots because they're not going to respond.

Nicole Costa, 17, of Glen Head, N.Y., took growth hormone for seven years starting at age 6. Her doctor had predicted she'd be only 4-feet-8; she's now 5-feet-2.

She recalls being ostracized in elementary school, unable to reach the water fountain and rejected for sports teams. Had she not grown, she said she couldn't have driven a regular-sized car or bought off-the-rack clothes.

"I hope ... this opportunity will be available to all the children now walking in the shoes I outgrew," she told the FDA panel. "It will make their world a different place."

Panelists backed the plan after hearing from Nicole.

To move from an adult height of 4-feet-10 to even a little over 5-feet can be significant, and is something families will have to decide for themselves, explained panel chairman Dr. Glenn Braunstein of the University of California, Los Angeles.

Lilly's criteria are slightly stricter than what pediatric groups consider abnormally shortness, as determined by comparison to average growth patterns.

Panelists demanded additional research on how best to use the drug, and that, if FDA approves the new use, Lilly start a mandatory registry to track every otherwise healthy child given the drug.

The FDA isn't bound by its advisers' recommendations but typically follows them.

Growth hormone already is approved to treat children suffering from a lack of that hormone or from certain genetic or kidney conditions that also stunt growth.

Harder to handle are children for whom doctors can find no disease making them significantly shorter -- 7 or 8 inches -- than their peers.

It's unfair to withhold a treatment that works from those children because doctors can't pinpoint a certain cause of their shortness, argued Lilly consultant Dr. Raymond Hintz of Stanford University.

Studies showed most patients got just a small benefit, an inch or two. But some had dramatically better results.