Improved treatment options for irritability and novel treatments for core symptoms of autism spectrum disorder (ASD) are the key unmet needs for the autism community, according to GlobalData.
GlobalData, a data and analytics company, conducted a survey which revealed treatment options to target irritability and aggression associated with autism, and the core symptoms of ASD, were ranked by high prescribing physicians as important in the ASD market.
While there are many different treatment options used to target the variety of associated symptoms, the majority of these are prescribed off-label, resulting in significant opportunity for developers in the autism market.
Pippa Salter, Senior Neurology Analyst at GlobalData, comments: “Any novel products for the treatment of symptoms associated with ASD, such as irritability and aggression, will have to demonstrate significantly improved efficacy and/or safety in order to displace the widely available cheap generic therapies currently used, such as atypical antipsychotics, antidepressants, and stimulants. In contrast, GlobalData expects that any products developed for the core symptoms of ASD, which include social and communication impairments, and repetitive behaviors, would likely see strong uptake and would dramatically alter the ASD market.”
Irritability associated drugs rarely prescribed for children
Of the symptoms associated with autism spectrum disorder, experts interviewed by GlobalData agreed autistic people were most likely to seek treatment for irritability and aggression. Although there are two treatments for irritability associated with ASD approved by the Food and Drug Administration (FDA): Johnson & Johnson’s Risperdal (risperidone) and Otsuka Pharmaceutical’s Abilify (aripiprazole), these can have negative side effects and are not often prescribed for young children.
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“For irritability and aggression, significant opportunity remains for treatment options with improved safety profiles as well as strong efficacy. To this end, Otsuka and AbbVie are investigating their second-generation atypical antipsychotic products, Rexulti (brexpiprazole) and Vraylar (cariprazine), respectively, for ASD. Additionally, there are several novel mechanisms of action being trialed as alternatives to the atypical antipsychotic class, including Jazz Pharmaceuticals’ cannabinoid receptor 1 agonist GWP-42006 (cannabidivarin), and Axial Therapeutics’ gut-restricted molecular therapy AB-2004. Of these novel options, KOLs thought that AB-2004 looked to be a particularly promising alternative to the atypical antipsychotics used for irritability,” Salter added.
No approved therapies for core ASD symptoms
There are no approved therapies to target the core symptoms of autism and therefore, each of these symptoms individually constitutes an unmet need for therapeutic intervention. Autism experts interviewed by GlobalData also pointed to the lack of treatment options for core symptoms as the primary and most significant unmet need for autistic individuals.
“There are several novel pipeline products aiming to overcome this particular unmet need. These include Johnson & Johnson’s fatty acid amide hydrolase inhibitor JNJ-5279, Yamo Pharmaceuticals’ tyrosine 3 monooxygenase inhibitor metyrosine, and Paxmedica’s purinergic receptor P2X and P2Y antagonist PAX-101 (suramin hexasodium),” Salter continued.
“All of these products are yet to progress to Phase III clinical trials, so it will be several years before any therapies targeting the core symptoms could enter the market.”