The Food and Drug Administration (FDA) has approved two drugs for treating irritability associated with the autism (risperidone and aripiprazole). The prevalent behavioral problems associated with autism spectrum disorder, including repetitive behavior, communication, and social issues, have not been able to be improved by medication at this time, since there are no drugs are currently approved to address these core symptoms.

However, a breakthrough may be on the horizon. A major Swiss pharmaceutical company, Roche, says it has received a designation from the Food and Drug Administration to help expedite what could be the first drug to treat these core characteristics of autism. Roche released news in January 2018 that the FDA has granted its breakthrough therapy designation for the development of balovaptan, a drug that potential to improve “core social interaction and communication” in those with autism. Results from a clinical trial in adults with autism released in 2017 indicate that balovaptan was successful in helping to improve challenging social behaviors. Additionally, it was deemed safe and well tolerated.

Another trial looking at children and adolescents on the spectrum is underway and additional studies are in the works. Improving these problems could help a person with autism spectrum disorder function more effectively in all areas of their lives. However, risperidone and aripiprazole can actually ease these core symptoms, because relieving irritability often improves sociability while reducing tantrums, aggressive outbursts and self-injurious behaviors.

Both risperidone (Risperdal) and aripiprazole (Abilify) were approved by the Food and Drug Administration (FDA) for autism-related irritability. These two drugs are in a class known as atypical antipsychotics and are believed to produce better results than previously used “typical” antipsychotics. In addition to addressing irritability, these drugs also may reduce behaviors such as aggression, deliberate self-injury, and “lashing out” or temper tantrums. The drugs address these behaviors about 30 to 50 percent of the time, but don’t address all behavior issues — and psychiatric problems are common in children with autism.

Other atypical antipsychotics that have been studied recently with encouraging results are olanzapine (Zyprexa®) and ziprasidone (Geodon®). Ziprasidone has not been associated with significant weight gain, though some side effects of these medications can include increased appetite and weight gain. It is important to consult with your doctor to monitor these side effects, and also make a commitment to a healthy diet and exercise.

The medications used for autism spectrum disorder may be used to treat similar symptoms in other disorders. Many of these medications are prescribed “off-label.” This means they have not been officially approved by the U.S. Food and Drug Administration (FDA) for use in children, but the doctor prescribes the medications if he or she feels they are appropriate for your child. Further research needs to be done to ensure not only the efficacy but the safety of psychotropic agents used in the treatment of children and adolescents.

Olanzapine (Zyprexa) and other antipsychotic medications are used “off-label” for the treating symptoms such as aggression in addition to other serious behavioral disturbances in children, including children with autism. Other medications are used to address symptoms or other disorders in children with autism. Fluoxetine (Prozac) and sertraline (Zoloft) are approved by the FDA for children age 7 and older with obsessive-compulsive disorder. Fluoxetine is also approved for children age 8 and older for the treatment of depression.

Two SSRIs, Fluoxetine and Sertraline, have been approved by the FDA to treat obsessive compulsive disorder (OCD) in children who are also diagnosed with autism spectrum disorder. Fluoxetine has been used to treat children with major depressive disorder (MDD) and OCD for over 14 years in the USA, has recently been expanded to other behavior disorders, including attention deficit hyperactivity disorder (ADHD), anxiety, and autism. Sertraline was approved by the FDA for children age 7 and older with obsessive-compulsive disorder. Despite the relative safety and popularity of SSRIs and other antidepressants, some studies have suggested that they may have unintentional effects on some people, especially adolescents and young adults.

The FDA adopted a “black box” warning label to be noted on all antidepressant medications about the potential increased risk of suicidal thinking or attempts in children and adolescents taking antidepressants. In 2007, the agency extended the warning to include young adults up to age 25. A “black box” warning is the most serious type of warning on prescription drug labeling. It states that patients of all ages should be closely monitored, especially in the beginning phases of treatment, if depression is worsening, or if there is suicidal thinking or behavior. If there are unusual changes in behavior such as sleeplessness, agitation, or withdrawal from normal social situations, it is important to notify your physician.

Typically developing children and children with autism may respond differently to certain medications. It is important that parents work with a doctor who has experience with children with autism. Children taking any medications should be monitored closely. The doctor will prescribe the lowest dose possible to be effective. Ask the doctor about any side effects the medication may have and keep a record of how your child responds to the medication. It will be helpful to read the “patient insert” that comes with your child’s medication. Some people keep the patient inserts in a small notebook to be used as a reference. This is most useful when several medications are prescribed.

The selective serotonin reuptake inhibitors (SSRI’s) are the drugs most often used for anxiety, depression, and/or obsessive-compulsive disorder (OCD) symptoms. The only SSRI approved by the FDA for both OCD and depression in children age 7 and older is fluoxetine, (Prozac®). FDA-approved me for OCD are fluvoxamine (Luvox®), age 8 and older; sertraline (Zoloft®), age 6 and older; and clomipramine (Anafranil®), age 10 and older. Using any of these medications help reduce repetitive behaviors and helps to increase frequency in eye contact and social contacts. The FDA is studying and analyzing data to better understand how to use the SSRI’s safely, effectively, and at the lowest dose possible.

Seizures occur in one in four persons with autism spectrum disorders (ASD), most often in those who have low IQ or are mute. They are treated with one or more of the anticonvulsants, including carbamazepine (Tegretol®), lamotrigine (Lamictal®), topiramate (Topamax®), and valproic acid (Depakote®). The level of the medication in the blood should be monitored carefully and adjusted so that the least amount possible is used to be effective. Although medication usually reduces the number of seizures, it cannot always eliminate them.

Stimulant medications such as methylphenidate (Ritalin®), used safely and effectively in persons with attention deficit hyperactivity disorder, have also been prescribed for children with autism. These medications may decrease impulsivity and hyperactivity in some children, especially those higher functioning children.

Several other medications have been used to treat ASD symptoms; among them are other antidepressants, naltrexone, lithium, and some of the benzodiazepines such as diazepam (Valium®) and lorazepam (Ativan®). The safety and efficacy of these medications in children with autism has not been proven. Since people may respond differently to different medications, your child’s unique history and behavior will help your doctor decide which medication might be most beneficial.