Abstract
Overview
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.
Report 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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