Use of Drug OKd for Premature Babies
WASHINGTON — The Food and Drug Administration said Wednesday that it has authorized widespread use of an experimental drug to treat hyaline membrane disease, a serious respiratory ailment that is one of the leading causes of death and disability in premature infants.
In 1963, the disease killed 2-day-old Patrick Bouvier Kennedy, a baby boy born to President and Mrs. John F. Kennedy.
The condition, also known as respiratory distress syndrome, afflicts about one in five premature infants in the United States. It is caused when a premature infant is unable to produce a naturally occurring substance that coats the lungs and keeps them from collapsing when the infant exhales.
Not Yet Approved
The drug, a synthetic surfactant, or surface-acting agent, has not yet been approved for marketing. It is being made available under the investigational new drug status, informally known as “compassionate use.”
“When there is adequate evidence that a drug is effective and safe for a serious or life-threatening disease, and when no satisfactory alternative exists, we can make the drug available,” FDA Commissioner Frank E. Young said in a statement.
The manufacturer, Burroughs Wellcome Co. of Research Triangle Park, N.C., said that it will make the drug available free of charge to hospitals nationwide.
The drug, called Exosurf, was invented by Dr. John Clements of UC San Francisco. It was shown in clinical studies to reduce by 33% to 40% the number of deaths in infants who either had hyaline membrane disease or were at risk of developing it, according to the company.
The trials, which began in 1986, were conducted in 50 hospitals in the United States and Canada, including Cedars-Sinai Medical Center and Women’s Hospital in Los Angeles, Children’s Hospital in San Francisco and UC Davis. The tests involved about 3,000 premature infants.
Ron Wise, a spokesman for Cedars-Sinai Medical Center, said the hospital had only recently entered the studies and had used the drug on only about seven babies. Based on that limited experience, however, “the initial impression was positive and the babies seemed to be responding better than would be expected without the drug,” Wise said.
The condition affects as many as three in four babies born at less than 30 weeks gestation or who weigh less than 2.75 pounds, the company said. Of the 230,000 to 250,000 premature infants born each year, approximately 40,000 to 50,000 develop the syndrome and 5,000 die.
The studies were conducted on premature babies weighing between 1 and 1.5 pounds. The drug was not tested on larger babies because those weighing more than 2.5 pounds develop the disease much less frequently, according to the manufacturer.
Conventional treatment for the condition usually involves placing infants on mechanical ventilators that force high concentrations of oxygen into the lungs. High ventilator pressures and high concentrations of oxygen can by themselves damage the lungs, the company said.
Months of Treatment
Some premature babies with hyaline membrane disease require several months of treatment in an intensive care nursery with ventilators and oxygen before they can breathe on their own, according to Burroughs.
The drug is a soapy, film-like substance that lines the lungs, reducing surface tension. This enables the lungs to inflate easily and prevents them from collapsing during exhalation, Burroughs said. The drug is administered through a tube into the lungs, and premature infants must be on a mechanical ventilator to receive it, the FDA said. But company executives said that, coupled with use of the drug, less ventilator pressure was necessary and infants could be removed from the ventilator earlier.
