Product Profiles: Autism Spectrum Disorders – Growing interest in underserved market

Over the past 6 years, three major events have impacted the autism spectrum disorders market. In 2006, Risperdal became the first drug to receive approval from the US FDA for the symptomatic treatment of irritability in children and adolescents with ASDs. Later, in 2009, the FDA approved Abilify for the same indication. In 2008, generic risperidone launched following Risperdal’s US patent expiry.

Features and benefits

• Overview of marketed and pipeline drugs in clinical development for autism spectrum disorders.
• Analysis of the market positioning of each product approved for the treatment of autism spectrum disorders.
• Assessment of the strengths, weaknesses, opportunities, and threats for marketed and late-stage pipeline candidates.
• Summary of the clinical and commercial attractiveness for each of the marketed products.
• Insight from key opinion leaders on marketed and pipeline products.

Highlights

Risperdal and Abilify are the only two products approved for the treatment of autism spectrum disorders (ASDs). Both of these atypical antipsychotics are indicated for the management of irritability symptoms associated with ASDs in the US. Due to the failure of these drugs to address the core symptoms of ASDs, many other drugs are used off-label.

Abilify is the most recent entrant to the ASDs market, having received US Food and Drug Administration (FDA) approval in November 2009. The drug's partial agonism of dopamine receptors unique mechanism of action is considered a key differentiating factor, producing a superior side-effect profile compared to other atypical antipsychotics.

To date, none of the four late-stage pipeline candidates for autism spectrum disorders has publically presented the late-stage Phase II or Phase III trial data necessary for the clinical assessment of these compounds.

Your key questions answered

• What are the key differentiating features of approved therapies for the treatment of autism spectrum disorders?
• How do key opinion leaders perceive available treatments for autism spectrum disorders?
• What opportunities are there for existing players in the autism spectrum disorders market to increase their market share?
• What is the development status of current pipeline candidates?
• What clinical trial designs are current pipeline candidates following? What do pipeline candidates need to demonstrate in order to be successful?

Table Of contents

OVERVIEW
Catalyst
Summary

EXECUTIVE SUMMARY
Update details: April 2012
Strategic scoping and focus
Datamonitor key findings
Related reports

MARKET DEFINITION AND OVERVIEW
Market definition
Product overview

MARKETED PRODUCT PROFILES
Abilify (aripiprazole; Bristol-Myers Squibb/Otsuka Pharmaceuticals)
Drug profile
Development overview
SWOT analysis
Product positioning
Clinical and commercial attractiveness
Risperdal (risperidone; Johnson & Johnson)
Drug profile
Development overview
SWOT analysis
Product positioning
Clinical and commercial attractiveness

PIPELINE PRODUCT PROFILES
CM-AT (Curemark)
Drug profile
Development overview
SWOT analysis
AT001 (fluoxetine; Autism Therapeutics)
Drug profile
Development overview
SWOT analysis
Other drugs in clinical development for autism spectrum disorders
STX-209 (arbaclofen; Seaside Therapeutics)
Memantine hydrochloride (Forest Laboratories/Merz)
UCB-MNC (umbilical cord blood mononuclear stem cells; Beike Biotech)
Carbetocin (Kyalin Bioscience)
RG-7314 (Roche)
RO-5028442 (Roche)

BIBLIOGRAPHY
Journal papers
Websites
Datamonitor reports

APPENDIX
PharmaVitae Explorer database
Contributing experts
Conferences attended
Report methodology

List Of Tables

Table: Summary of therapeutic classes in autism spectrum disorders by A natomical Therapeutic Chemical code, 2012
Table: Summary of the ICD-10 codes used to derive drugs indicated and in clinical development for autism spectrum disorders
Table: Key marketed products for autism spectrum disorders, June 2012
Table: Key pipeline products for autism spectrum disorders, April 2012
Table: Abilify (aripiprazole; Otsuka/Bristol-Myers Squibb) – drug profile for autism spectrum disorders, 2012
Table: Overview of pivotal and long-term trials conducted for Abilify (aripiprazole) in autism spectrum disorders
Table: Overview of side effects associated with Abilify in two pivotal Phase III clinical trials in autism spectrum disorders
Table: Datamonitor drug assessment summary of Abilify (aripiprazole; Bristol-Myers Squibb/Otsuka Pharmaceuticals) for autism spectrum disorders, 2012
Table: Risperdal (risperidone; Johnson & Johnson) – drug profile for autism spectrum disorders, 2012
Table: Overview of pivotal trials conducted for Risperdal in the treatment of autism spectrum disorders
Table: Overview of side effects reported in two pivotal Phase III clinical trials of Risperdal in autism spectrum disorders
Table: Datamonitor drug assessment summary of Risperdal (risperidone; Johnson & Johnson) for autism spectrum disorders, 2011
Table: CM-AT (Curemark) – drug profile, 2012
Table: Phase III clinical trial of CM-AT in the treatment of core symptoms of autism spectrum disorders, 2012
Table: Phase III open-label extension study of CM-AT in the treatment of core symptoms of autism spectrum disorders, 2012
Table: AT001 (fluoxetine; Autism Therapeutics) – drug profile, 2012
Table: Overview of Phase III trials conducted for fluoxetine in the treatment of autism spectrum disorders
Table: STX-209 (arbaclofen; Seaside Therapeutics) – drug profile, 2012
Table: Memantine hydrochloride (Forest Laboratories/Merz) – drug profile, 2012
Table: Phase II clinical trial of memantine hydrochloride in the treatment of children with autism spectrum disorders, 2012
Table: An early Phase II clinical trial of memantine hydrochloride in the treatment of adults with autism spectrum disorders, 2012
Table: UCB-MNC (Beike Biotech) – drug profile, 2012
Table: Carbetocin (Kyalin Bioscience) – drug profile, 2012

List Of Figures

Figure: Abilify (aripiprazole; Otsuka Pharmaceuticals/Bristol-Myers Squibb) SWOT analysis in autism spectrum disorders, 2011
Figure: Datamonitor drug assessment summary of Abilify (aripiprazole; Bristol-Myers Squibb/Otsuka Pharmaceuticals) for autism spectrum disorders, 2012
Figure: Risperdal (risperidone; Johnson & Johnson) SWOT analysis in autism spectrum disorders, 2012
Figure: Datamonitor drug assessment summary of Risperdal (risperidone; Johnson & Johnson) for autism spectrum disorders, 2011
Figure: CM-AT (Curemark) SWOT analysis in autism spectrum disorders, 2012
Figure: AT001 (fluoxetine; Autism Therapeutics) SWOT analysis in autism spectrum disorders, 2012
Figure: Phase II clinical trial of STX-209 for autism spectrum disorders
Figure: Ongoing late Phase II clinical trial of STX-209 for autism spectrum disorders
Figure: The PharmaVitae Explorer