Autism Medication: What’s FDA-Approved and How Doctors Decide

There is no autism medication that treats autism itself. No current medicine changes core social communication differences [1]. Two medicines, risperidone and aripiprazole, are FDA-approved for severe irritability associated with autism, and doctors may also prescribe medicines for co-occurring ADHD, anxiety or sleep problems, after other causes and supports are considered [1][2][3].

What autism medication can and cannot do

The AAP is clear that no current medication corrects the core social and communication features of autism [1]. The UK’s NICE guideline says not to use antipsychotics, antidepressants or anticonvulsants to manage core features of autism in children [4].

Medicine may be considered to help with [1]:

• Co-occurring conditions, such as ADHD, anxiety or mood disorders.
• Behaviors or symptoms that cause significant distress or impairment, such as aggression, self-injury, sleep problems, severe mood swings or hyperactivity.

Any autism medication is most helpful as part of a wider plan that includes behavioral, educational and family support [1].

How doctors decide about autism medication

The AAP recommends that medication is only considered after [1]:

• Understanding the behavior. When did it start? What makes it worse? Could it be a way of communicating distress or refusal?
• Checking for medical causes. Pain from reflux, ear infections, dental problems or injuries can show up as aggression or self-injury.
• Trying behavioral support first, including a functional behavioral assessment at school and strategies for home.
• Shared decision-making with your family and your child, based on your goals and values.

The prescribing doctor should understand the medicine’s uses, risks, interactions and monitoring needs [1]. NICE recommends that antipsychotics for behavior that challenges are started by a pediatrician or psychiatrist, reviewed after 3 to 4 weeks, and stopped if there is no clinically important response at 6 weeks [4]. Your doctor will advise on the right medicine and dose for your child.

FDA-approved autism medication: risperidone and aripiprazole

Risperidone (brand name Risperdal) and aripiprazole (brand name Abilify) are the only medicines with FDA-approved labeling specific to irritability associated with autism [1].

• Risperidone is approved for irritability associated with autistic disorder, including aggression toward others, deliberate self-injury, temper tantrums and quickly changing moods. Effectiveness was shown in trials of children and teens aged 5 to 17 [2].
• Aripiprazole is approved for irritability associated with autistic disorder, with trials in children aged 6 to 17 [3].

How well do they work?

In a landmark 2002 trial of 101 autistic children with serious behavior problems, 69% improved substantially on risperidone over 8 weeks, compared with 12% on placebo. For two thirds of children who improved, the benefit lasted at least 6 months [5]. A Cochrane review of aripiprazole found moderate-quality evidence that it reduced irritability, hyperactivity and repetitive movements in the short term [6]. A meta-analysis of 46 trials found these two medicines had the strongest evidence for reducing irritability [7].

Common side effects

Like any autism medication, both medicines can cause significant side effects, so they are generally kept for situations where the benefit is likely to outweigh the risk.

• Weight gain and increased appetite. In the 2002 trial, children on risperidone gained an average of 2.7 kg in 8 weeks, compared with 0.8 kg on placebo [5]. The aripiprazole Cochrane review found an average extra gain of about 1.1 kg [6].
• Sleepiness, tiredness, dizziness and drooling [5][3].
• Tremor and movement side effects (called extrapyramidal symptoms) [6][3].
• Raised prolactin (risperidone). In short trials, 49% of children on risperidone had raised prolactin levels, compared with 2% on placebo [2].
• Changes in blood sugar and cholesterol can occur with this class of medicine [1][2].

The AAP recommends monitoring weight, height, BMI, blood sugar and cholesterol, and checking for movement side effects [1]. In one trial, the diabetes medicine metformin reduced weight gain caused by these medicines in autistic young people [8]. Ask your doctor what monitoring plan they will use.

Autism medication for ADHD symptoms

About half of autistic children may also meet criteria for ADHD [1]. The same medicines used for ADHD are used in autistic children, but monitoring is important because autistic children may be more sensitive to side effects [1].

• Stimulants (such as methylphenidate). A Cochrane review found low-quality evidence that methylphenidate improves hyperactivity in autistic children, with no evidence it worsens core autism features [9]. In a key trial, about half of children responded, but responses were smaller than in children with ADHD alone, and side effects led 18% to stop [10]. Common side effects include reduced appetite and trouble sleeping, and sometimes irritability or social withdrawal [1].
• Non-stimulants. Guanfacine (extended-release) improved hyperactivity in a trial of 62 autistic children, with 50% responding compared with 9% on placebo. Common side effects were drowsiness, tiredness and reduced appetite [11]. Atomoxetine and clonidine are other options [1]. Guanfacine and clonidine can lower blood pressure and heart rate and should not be stopped suddenly [1].

Before medicine, the AAP recommends checking that schoolwork and instructions match your child’s language and learning level, since inattention can come from tasks a child cannot yet understand [1]. See our guide to IEPs and 504 plans for autism.

Anxiety, repetitive behaviors and sleep

SSRIs (such as fluoxetine or sertraline) are antidepressants sometimes used for anxiety in autistic children. The AAP notes that studies have not shown SSRIs work for repetitive behaviors related to autism, though they may help anxiety. Restlessness or over-activation is a side effect that sometimes leads to stopping them [1]. A 2019 trial of fluoxetine for obsessive-compulsive behaviors in autistic young people had mixed results [12]. For anxiety, cognitive behavioral therapy is the first-line treatment [1].

Sleep. Melatonin has evidence for improving sleep in autistic children [1]. NICE recommends trying a sleep plan first, and using melatonin only with specialist advice [4]. See our guides to autism sleep problems and melatonin for autism.

What about leucovorin?

You may have seen news about leucovorin (folinic acid) for autism. In March 2026, the FDA approved leucovorin tablets for cerebral folate deficiency in people with a confirmed variant in the FOLR1 gene, an extremely rare genetic condition [13]. It is not approved to treat autism. In October 2025, the AAP said it does not recommend routine use of leucovorin for autistic children, because the evidence is too limited, and that families who ask should have a shared discussion of risks and benefits with their doctor [14].

When to talk to a professional

Talk to your child’s doctor if your child’s behavior is putting them or others at risk, if distress is severe or getting worse, or if sleep, anxiety or attention problems are affecting daily life. Seek urgent help for serious self-injury or if anyone is in danger. If your child already takes autism medication, ask for regular reviews, and never stop or change a medicine without medical advice. Our medical disclaimer explains how to use this guide.

Frequently asked questions

Is there a medication that treats autism?

No. No current medicine changes the core social and communication features of autism. Medicines may help with specific problems that often occur alongside autism, such as severe irritability, ADHD symptoms, anxiety or sleep difficulties. They work best as part of a broader plan that includes behavioral, educational and family support.

What is risperidone used for in autism?

Risperidone is FDA-approved to treat irritability associated with autism in children and teens, including aggression, deliberate self-injury, severe tantrums and quickly changing moods. It can work well for some children, but common side effects include weight gain, increased appetite, sleepiness and raised prolactin, so regular monitoring by your doctor is essential.

Can autistic children take ADHD medication?

Yes. Many autistic children also have ADHD, and the same medicines are used. Methylphenidate and guanfacine have trial evidence for reducing hyperactivity in autistic children. Responses may be smaller and side effects more common than in ADHD alone, so doctors usually start carefully and monitor closely. Your doctor will advise on the right medicine and dose.

Is leucovorin approved for autism?

No. In March 2026, the FDA approved leucovorin for cerebral folate deficiency caused by a confirmed FOLR1 gene variant, which is extremely rare. It is not approved for autism. The American Academy of Pediatrics does not recommend routine leucovorin use for autistic children and advises shared decision-making with your doctor.

References

1. Hyman SL, Levy SE, Myers SM; AAP Council on Children With Disabilities, Section on Developmental and Behavioral Pediatrics. Identification, evaluation, and management of children with autism spectrum disorder. Pediatrics. 2020;145(1):e20193447. Link
2. Janssen Pharmaceuticals. RISPERDAL (risperidone) prescribing information. DailyMed, US National Library of Medicine. Label revised 2026. Link
3. Otsuka America Pharmaceutical. ABILIFY (aripiprazole) prescribing information. DailyMed, US National Library of Medicine. Label revised 2025. Link
4. National Institute for Health and Care Excellence. Autism spectrum disorder in under 19s: support and management. Clinical guideline CG170. Published August 2013; last updated June 2021. Link
5. McCracken JT, McGough J, Shah B, et al; Research Units on Pediatric Psychopharmacology Autism Network. Risperidone in children with autism and serious behavioral problems. N Engl J Med. 2002;347(5):314-321. Link
6. Hirsch LE, Pringsheim T. Aripiprazole for autism spectrum disorders (ASD). Cochrane Database Syst Rev. 2016;(6):CD009043. Link
7. Fung LK, Mahajan R, Nozzolillo A, et al. Pharmacologic treatment of severe irritability and problem behaviors in autism: a systematic review and meta-analysis. Pediatrics. 2016;137(Suppl 2):S124-S135. Link
8. Anagnostou E, Aman MG, Handen BL, et al. Metformin for treatment of overweight induced by atypical antipsychotic medication in young people with autism spectrum disorder: a randomized clinical trial. JAMA Psychiatry. 2016;73(9):928-937. Link
9. Sturman N, Deckx L, van Driel ML. Methylphenidate for children and adolescents with autism spectrum disorder. Cochrane Database Syst Rev. 2017;11:CD011144. Link
10. Research Units on Pediatric Psychopharmacology Autism Network. Randomized, controlled, crossover trial of methylphenidate in pervasive developmental disorders with hyperactivity. Arch Gen Psychiatry. 2005;62(11):1266-1274. Link
11. Scahill L, McCracken JT, King BH, et al. Extended-release guanfacine for hyperactivity in children with autism spectrum disorder. Am J Psychiatry. 2015;172(12):1197-1206. Link
12. Reddihough DS, Marraffa C, Mouti A, et al. Effect of fluoxetine on obsessive-compulsive behaviors in children and adolescents with autism spectrum disorders: a randomized clinical trial. JAMA. 2019;322(16):1561-1569. Link
13. US Food and Drug Administration. FDA approves first treatment for patients with cerebral folate transport deficiency. News release. March 10, 2026. Link
14. American Academy of Pediatrics. Interim guidance: use of leucovorin in autistic pediatric patients. October 31, 2025. Link

Professional review: this guide is waiting for review by a qualified clinician. It was written and checked against the published sources listed above. How we check our guides.

This guide is general education, not medical advice. Always talk to your child’s doctor or care team. Last checked: October 2026.