Irritability and new treatments for key symptoms most dissatisfied with autism

According to GlobalData, improved treatment options for irritability and new treatments for core symptoms of autism spectrum disorder (ASD) are the top unmet needs of the autism community.

GlobalData, a data and analytics company, conducted a survey that found that treatment options for targeting irritability and aggression associated with autism, and core symptoms of ASD, were ranked by 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, providing significant opportunities for developers in the autism market.

Pippa Salter, Senior Neurology Analyst at GlobalData, comments: “Any new product for the treatment of symptoms associated with ASD, such as irritability and aggression, will need to demonstrate significantly improved efficacy and/or safety in order to replace the cheap generic widely available. currently used therapies, such as atypical antipsychotics, antidepressants and stimulants. In contrast, GlobalData expects all products developed for the core symptoms of ASDs, which include social and communication disorders, and repetitive behaviors, to experience high adoption and significantly change the ASD market.

Drugs associated with irritability rarely prescribed to children

Of the symptoms associated with autism spectrum disorders, experts surveyed by GlobalData agreed that people with autism were most likely to seek treatment for irritability and aggression. Although there are two Food and Drug Administration (FDA)-approved treatments for ASD-associated irritability: Johnson & Johnson’s Risperdal (risperidone) and Otsuka Pharmaceutical’s Abilify (aripiprazole), these can have negative side effects and are not often prescribed to young children. .

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“For irritability and aggression, significant opportunities remain 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 In addition, several new mechanisms of action are being tested as alternatives to the class of atypical antipsychotics, including the cannabinoid 1 receptor agonist GWP-42006 (cannabidivarin) of Jazz Pharmaceuticals and Axial Therapeutics’ bowel-restricting molecular therapy AB-2004 Among these new options, KOLs believed AB-2004 appeared to be a particularly promising alternative to 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 polled by GlobalData also highlighted the lack of treatment options for core symptoms as the number one and most important unmet need for people with autism.

“There are several new pipeline products aimed at overcoming this particular unmet need. These include the fatty acid amide hydrolase inhibitor JNJ-5279 from Johnson & Johnson, the tyrosine 3 monooxygenase inhibitor metyrosine from Yamo Pharmaceuticals, and the purinergic receptor antagonist P2X and P2Y PAX-101 (hexasodium suramin) from Paxmedica,” continued Salter.

“All of these products have yet to go through phase III clinical trials, so it will be several years before therapies targeting core symptoms can enter the market.”

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