YSN and Child Study Center Research Contributes toFDA Approval of Risperidone for Autism
New Haven, CT — November 2, 2006
The U.S. Food and Drug Administration has given approval to market the antipsychotic drug risperidone for the treatment of children with autism accompanied by tantrums, aggression and self-injurious behavior. The medication can be prescribed for children ages 5 to 16 to relieve these signs of autism, the FDA announced in October.
This is the first time the FDA has approved any medication for use in children and adolescents with autism. The approval was based on the results of a multi-site study conducted by the NIMH-funded Research Units of Pediatric Psychopharmacology (RUPP) Autism Network at five academic centers: Johns Hopkins, Indiana University, Ohio State University, UCLA and Yale.
"This is an exciting development and it is a first for the field of child psychiatry," remarked Lawrence Scahill, PhD, MSN, who was one of the principal investigators for the study. He added, "The FDA approval was based in large part on a federally funded study -- rather than an industry-sponsored study -- so this approval is a welcomed translation of clinical research to policy." Dr. Scahill is a Professor of Nursing and Child Psychiatry at Yale and the Director of the RUPP Autism Network at the Yale Child Study Center. He is also Acting Associate Dean for Scholarly Affairs at Yale School of Nursing.
About 1.5 million Americans have autism, a chronic developmental disorder that appears in early childhood. It is characterized by profound impairments in the ability to relate to others, delayed language, and restricted patterns of behavior. It affects as many as 20 children per 10,000. Although causes of autism are unknown for most cases, available evidence implicates abnormalities in brain development. Twin and family studies indicate a strong genetic contribution, though specific causative genes have not been identified.
"This approval should benefit many autistic children as well as their parents and other care givers," according to Steven Galson, M.D., director of the FDA's Center for Drug Evaluation and Research."Our agency strongly encourages the development of appropriate pediatric labeling for adult drugs, and risperidone is a welcome addition to the growing number of such products that have been shown to have an appropriate risk-benefit profile when tested in children."
"Despite the hints of other benefits from risperidone, the primary target for the medication is the moderate to severe behavioral problems that occur in some children with autism," noted Dr. Scahill. He emphasized, "This is an important point, because risperidone is a potent drug that is appropriate for treating serious behavioral problems -- not autism per se."
As with many other medications, risperidone has been prescribed "off label" for the treatment of children with autism for many years. The RUPP study showed that risperidone was superior to placebo for reducing aggression and explosive behavior. After eight weeks of treatment, children who were treated with risperidone showed a 50% improvement in tantrums, aggression and self-injury compared to little or no benefit for the placebo group. This report was published in the August 2002 edition of the New England Journal of Medicine. Since then, the RUPP Network has published several other reports showing that risperidone may also have beneficial effects other outcomes such as repetitive behavior and hyperactivity.
In July 2005, findings by the same investigators showed that positive effect of risperidone on serious behavioral problems endured over time and the drug was well tolerated for up to six months in the treatment of children with autism. The study also showed that it was not necessary to increase the medication dose to maintain these improvements over the six month observation period. According to Dr. Scahill, "Our findings broke new ground in the treatment of autism. For the first time, we have data on both the short- and longer-term benefits of medication for children with autism."
Health insurers often limit prescription-drug coverage to uses of medications specified in the FDA's approvals, which define how companies can market a treatment. Previously, risperidone was only approved for use as an antipsychotic and patients with autism did not receive coverage. "Insurance companies had argued that efficacy was not proven," said Peter Bell, president and chief executive officer of Cure Autism Now, an advocacy group which funds autism research. "Now that risperidone is approved for autism, this won't happen."
"Having FDA approval means physicians will have clear instructions on the label for how to use risperidone for autism symptoms. And this means a lot to patients, and their families," said Bell.
