Alpha 1 Antitrypsin Deficiency: Key Trends and Statistics Just Published

Wed Feb 20, 2008 5:50am EST

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   Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency

   To order this report:

   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.

   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 usesGain an understanding
of some of the principal challenges in the treatment of alpha 1
antitrypsin deficiency as defined by key opinion leaders.

   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.

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


   About the Respiratory & Infectious Disease (RID) analysis team 2


   Scope of the analysis 3

   Datamonitor insight into the disease market 4

   Related reports 4


   Alpha-1 antitrypsin deficiency is a hereditary disease found
mainly in Caucasians 6

   Etiology of alpha-1 antitrypsin deficiency 6

   Genetics of Alpha-1 antitrypsin deficiency 8

   Epidemiology 10

   Emphysema 12

   Liver disease 14

   Unmet needs 17

   Risk factors 17

   Smoking 18

   Environmental tobacco smoke exposure 19

   Occupational exposure 19

   Bacterial infections 20

   Body mass index 20


   Diagnosis of alpha-1 antitrypsin deficiency 21

   Lung function 21

   Imaging 21

   Serum alpha-1 antitrypsin levels 22

   Biochemical markers 22

   Phenotyping 22

   Diagnostic guidelines 22

   Genetic screening 23

   Increase in age at diagnosis and delay in diagnosis of alpha-1
antitrypsin deficiency 25

   Problems with physician knowledge 27

   Difficulty in conducting clinical trials in COPD 27

   Declining smoking rates 28


   Standard COPD therapy 29

   Augmentation therapy 29

   Therapeutic rationale 29

   Market analysis 32

   Cost and reimbursement 33

   Antibiotic therapy 34

   Organ transplant 34

   Pulmonary rehabilitation, supplementary oxygen and genetic
counseling 35


   Inhaled alpha-1 antitrypsin augmentation therapy 36

   Arriva/Hyland 37

   Kamada 37

   Talecris 37

   Recombinant alpha-1 antitrypsin augmentation therapy 37

   Gamma retinoid agonists 38

   Gene therapy is in the far future 38

   Alpha-1 antitrypsin replacement therapy in cystic fibrosis 38

   Clinical trial endpoints 39

   Continuing medical education 39

   The role of patient support groups 40

   Neonatal genetic screening 42

   Transfer of treatments to the general emphysema population 43


   Articles 44

   Websites 49

   List of Tables

   Table 1: Alpha-1 antitrypsin levels in common genotypes 9

   Table 2: Estimated prevalence of the five main phenotypes of
alpha-1 antitrypsin deficiency in selected countries 11

   Table 3: Estimated numbers of each of the five main phenotypes of
alpha-1 antitrypsin deficiency in selected countries 12

   Table 4: Classification of recommendations for genetic testing 24

   Table 5: Comparison of augmentation therapies in the US 30

   List of Figures

   Figure 1: Alpha-1 antitrypsin production and activity 8

   Figure 2: An example of three-generation pedigree with alpha-1
antitrypsin deficiency 10

   Figure 3: Estimated numbers of PiZZ individuals in selected
European countries 13

   Figure 4: Liver disease in PiZZ patients by age 15

   Figure 5: Association between liver dysfunction and age in PiZZ
infants 16

   Figure 6: Flow diagram of anticipated liver disease outcomes in
PiZZ infants 17

   Figure 7: Decline in lung function by smoking status 19

   Figure 8: Hospital admissions for alpha-1 antitrypsin deficient
patients in Norway, 2005 26

   Figure 9: SWOT analysis of human, plasma derived augmentation
therapy 33

   Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency

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