Introduction

Despite being one of the most common hereditary diseases among Caucasians, low physician awareness and a lack of disease-modifying drugs have led to massive underdiagnosis of the condition. the resulting perceived small number of patients and restricted market is a major obstacle for the development of new treatments.

Scope

Understand the epidemiology of alpha-1 antitrypsin deficiency and assess the untapped patient potential. Evaluate current strategies for the diagnosis and treatment of this disease. Assess the potential for new drugs and their potential for additional uses Gain an understanding of some of the principal challenges in the treatment of alpha 1 antitrypsin deficiency as defined by key opinion leaders.

Highlights

Prevalence estimates have identified 200,000 heterozygous patients in the US and Europe making alpha-1 antitrypsin deficiency one of the most common hereditary disorders in the Western world. However, physician resistance has led to the diagnosis of only a small proportion of the estimated population. Enzyme replacement augmentation therapy is available but lacks randomized, controlled efficacy data. Although widely used in the US, augmentation therapy is unavailable in the UK and Denmark. Patients who receive standard COPD treatment in the UK have comparable mortality rates to US patients who receive augmentation therapy. Inhaled alpha-1 antitrypsin therapy holds promise for the prevention of disease progression but a lack of efficacy data and drug delivery issues are hindering development. Roche has a gamma retinoid agonist, R-667, in Phase II trials and the drug is the only potential therapy that offers lung regeneration through the promotion of alveolar growth.

Reasons to Purchase

Get an overview of alpha-1 antitrypsin deficient phenotypes and their prevalence in the US and Western European markets Assess the market opportunity, where established therapies and other developmental compounds fail to address key unmet clinical needs Enhance your commercial positioning through an improved understanding of the alpha-1 antitrypsin deficiency market dynamics.

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