ASD is highly heterogeneous, with wide variation in severity, co-occurring conditions and age of diagnosis, which complicates clinical trial design, regulatory approval and the development of broadly effective treatments. The neurobiological underpinnings of ASD remain poorly understood and no validated biomarkers exist to guide diagnosis and disease classification. Studies rely heavily on behavioral assessments to evaluate efficacy, many of which are inherently subjective and introduce additional variability into clinical outcomes. 

There are only two medications approved for ASD: Johnson & Johnson’s Risperdal (risperidone) and Otsuka’s Abilify (aripiprazole), which are highly generalized atypical antipsychotics. Both are approved to treat irritability associated with ASD and have generally comparable levels of efficacy and tolerability. No pharmacological therapies are available to address the core symptoms of ASD, and treatment of these symptoms relies on behavioral and psychosocial interventions. As a result, a substantial unmet medical need remains. Pipeline therapies aim to address these unmet needs, but none have emerged as particularly promising based on current data. 

This Datamonitor Healthcare report contains a Disease Analysis module.