HHS Secretary Louis W. Sullivan, M.D., today announced that the Food and Drug Administration has approved the use of the AIDS drug zidovudine, commonly called AZT, to treat children.
The new labeling provides dosage recommendations for patients 3 months to 12 years of age who have AIDS or symptoms of advanced infection with the AIDS virus.
FDA's approval of AZT for pediatric use means that the drug is now officially considered to be a standard therapy for children with AIDS. "This status should encourage more insurance providers to reimburse for zidovudine's use in pediatrics," Dr. Sullivan said.
Pediatric trials of AZT began in 1986 and have involved more than 200 children. They were sponsored by Burroughs Wellcome Company of Research Triangle Park, N.C., and conducted by researchers at the National Cancer Institute and through NIAID's AIDS Clinical Trials Group network. These data, along with data from studies of AZT in adults infected with the AIDS virus provided the basis for extending the labeling instructions to children.
FDA's Antiviral Drug Products Advisory Committee, a committee of outside experts, recommended the pediatric labeling by a vote of 7 to 1 on March 30.
Zidovudine, approved in 1987 for the treatment of patients age 13 and older with AIDS and symptoms of infection with the AIDS virus, was recently also approved for the treatment of patients with less advanced disease who have evidence of immunosuppression.
While there is no prohibition against physicians prescribing approved drugs for other patient populations, many physicians have been reluctant to use zidovudine in children because of concerns that the drug's side-effects might be more severe in them than in other patients. Clinical data now indicate that children receiving zidovudine experience side-effects similar to those that occur in adults at a similar stage of the disease.
Since last October, zidovudine has been distributed to physicians requesting it for children with AIDS or advanced AIDS virus infection under a Treatment IND (investigational new drug application) sponsored by Burroughs Wellcome, with medical and technical assistance from NIAID.
The Treatment IND mechanism was established by FDA to allow patients with serious or life-threatening conditions for which there are no satisfactory treatments to obtain promising experimental drugs.
Although zidovudine is the only drug shown effective in prolonging the lives of people with AIDS, it has significant side-effects. The drug can inhibit the production of red blood cells and may reduce white blood cell counts to the point where the drug has to be discontinued to avoid infections.
In January, the recommended dose of zidovudine for adults was lowered after clinical trials showed the lower dose to be as effective as standard higher dose regimens in prolonging life and slowing the development of symptoms, and was less likely to produce severe adverse reactions. The currently recommended dose for children may be lowered as a result of a clinical trial now underway.