What medications are available to help alleviate symptoms in children and adolescents with autism spectrum disorder (ASD)?

Medications Available for Autism Spectrum Disorder

There are currently 2 FDA-approved medications for treating symptoms associated with autism spectrum disorder (ASD): risperidone and aripiprazole, both approved specifically for irritability in children and adolescents aged 5-17 years. 1, 2

FDA-Approved Medications

Atypical Antipsychotics for Irritability

• Risperidone is FDA-approved for irritability associated with ASD in children aged 5-16 years, with an effective dose range of 0.5-3.5 mg/day (mean effective dose 1.16-1.9 mg/day). 1, 2
• Aripiprazole is FDA-approved for irritability associated with ASD in children and adolescents aged 6-17 years, with a dose range of 5-15 mg/day. 1
• Both medications significantly improve scores on the Aberrant Behavior Checklist Irritability subscale, targeting aggression, self-injury, temper tantrums, and mood lability. 1, 2

Off-Label Medications with Evidence

For Hyperactivity and Inattention

• Methylphenidate (stimulant) shows efficacy in 49% of children with ASD versus 15.5% on placebo, started at 0.3-0.6 mg/kg/dose, 2-3 times daily. 1, 3
• Extended-release guanfacine and extended-release clonidine (alpha-2 agonists) can be considered when stimulants are ineffective or not tolerated. 3

For Repetitive Behaviors

• SSRIs (selective serotonin reuptake inhibitors) have shown some benefit for repetitive behaviors, though evidence is limited. 1
• Fluvoxamine demonstrated statistically significant decrease in repetitive behaviors on the CY-BOCS Compulsions scale at doses of 2.4-20 mg/day. 1

For Sleep Disturbances

• Melatonin is often used as first-line treatment for sleep disturbances in ASD. 1
• Sedating antihistamines may improve sleep quality but have limited evidence for efficacy. 1

Total Count of Medications

In practical terms, there are approximately 7-8 distinct medication classes or specific agents with at least some evidence for helping symptoms in ASD:

1. Risperidone (FDA-approved) 1, 2
2. Aripiprazole (FDA-approved) 1
3. Methylphenidate 1, 3
4. Extended-release guanfacine 3
5. Extended-release clonidine 3
6. SSRIs (as a class, including fluvoxamine) 1
7. Melatonin 1

Critical Clinical Context

• No medications treat the core social communication deficits of ASD—all approved and studied medications target associated symptoms or comorbid conditions rather than core autism features. 1
• Medication should never substitute for appropriate behavioral and educational services, and combining medication with parent training is moderately more efficacious than medication alone. 1, 4
• The heterogeneity of ASD presentation means that children with ASD are more vulnerable to side effects than typically developing peers. 5
• Risperidone and aripiprazole remain the only medications with robust, replicated evidence from large randomized controlled trials, though their use is limited by significant side effects including weight gain, metabolic changes, and sedation. 6, 5, 7