Improved irritability treatments and novel treatments for ASD core symptoms are most important unmet needs in ASD market in 7MM, finds GlobalData

Improved treatments options for irritability and novel treatments for core symptoms of
autism spectrum disorder (ASD) are the most important unmet needs in the ASD market in the seven major markets (7MM*), finds GlobalData, a leading data and analytics company.

A recent survey** by GlobalData has revealed that treatment options to target irritability and aggression associated with ASD, and the core symptoms of ASD, were consistently ranked by high prescribing physicians as the two most important in the ASD market (ranking from 1 to 9, where 1 indicated the most significant need and 9 the least significant). This trend was seen throughout the 7MM.

ASD is a neurodevelopmental disorder characterized by varying social and communication impairments and pervasive repetitive behaviors. It is a highly heterogenous disorder with a wide range of associated non-core symptoms and severities. 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 ASD 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.”

Of the symptoms associated with ASD, key opinion leaders (KOLs) interviewed by GlobalData agreed that irritability and aggression were the most common symptoms that patients and/or their caregivers were likely to seek treatment for. Although there are two treatments for irritability associated with ASD approved by the Food and Drug Administration (FDA) that include Johnson & Johnson’s Risperdal (risperidone) and Otsuka Pharmaceutical’s Abilify (aripiprazole), these atypical antipsychotics are associated with undesirable side effects such as weight gain, and KOLs are reluctant to prescribe them in young children.

Salter continues: “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.”

There are currently no approved therapies to target the core symptoms of ASD and therefore, each of these symptoms individually constitutes an unmet need for therapeutic intervention, warranting targeted drug development. KOLs interviewed by GlobalData also identified the lack of treatment options for core symptoms as the primary and most significant unmet need for ASD patients.

Salter adds: “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). 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; however, KOLs highlighted JNJ-5279 as particularly interesting.”

* 7MM: US, France, Germany, Italy, Spain, UK, and Japan.

** GlobalData’s 2022 June autism spectrum disorder survey, sample size of 117 high-prescribing psychiatrists, pediatricians, and primary care physicians, who represented the 7MM.

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